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THE SCIENCE AND ETHICS OF 

GENETICALLY ENGINEERED HUMAN DNA 

HEARING 

BEFORE THE

SUBCOMMITTEE ON RESEARCH & TECHNOLOGY 

COMMITTEE ON SCIENCE, SPACE, AND 

TECHNOLOGY 

HOUSE OF REPRESENTATIVES 

ONE HUNDRED FOURTEENTH CONGRESS 

FIRST SESSION 

JUNE 16, 2015 

Serial No. 114–24 

Printed for the use of the Committee on Science, Space, and Technology 

( 

Available via the World Wide Web: http://science.house.gov 
(II) 

COMMITTEE ON SCIENCE, SPACE, AND TECHNOLOGY 

HON. LAMAR S. SMITH, Texas, Chair 

FRANK D. LUCAS, Oklahoma 

F. JAMES SENSENBRENNER, JR., 

Wisconsin 

DANA ROHRABACHER, California 

RANDY NEUGEBAUER, Texas 

MICHAEL T. MCCAUL, Texas 

MO BROOKS, Alabama 

RANDY HULTGREN, Illinois 

BILL POSEY, Florida 

THOMAS MASSIE, Kentucky 

JIM BRIDENSTINE, Oklahoma 

RANDY K. WEBER, Texas 

BILL JOHNSON, Ohio 

JOHN R. MOOLENAAR, Michigan 

STEVE KNIGHT, California 

BRIAN BABIN, Texas 

BRUCE WESTERMAN, Arkansas 

BARBARA COMSTOCK, Virginia 

DAN NEWHOUSE, Washington 

GARY PALMER, Alabama 

BARRY LOUDERMILK, Georgia 

RALPH LEE ABRAHAM, Louisiana 

EDDIE BERNICE JOHNSON, Texas 

ZOE LOFGREN, California 

DANIEL LIPINSKI, Illinois 

DONNA F. EDWARDS, Maryland 

SUZANNE BONAMICI, Oregon 

ERIC SWALWELL, California 

ALAN GRAYSON, Florida 

AMI BERA, California 

ELIZABETH H. ESTY, Connecticut 

MARC A. VEASEY, TEXAS 

KATHERINE M. CLARK, Massachusetts 

DON S. BEYER, JR., Virginia 

ED PERLMUTTER, Colorado 

PAUL TONKO, New York 

MARK TAKANO, California 

BILL FOSTER, Illinois 

SUBCOMMITTEE ON RESEARCH AND TECHNOLOGY

HON. BARBARA COMSTOCK, Virginia, Chair 

FRANK D. LUCAS, Oklahoma 

MICHAEL T. MCCAUL, Texas 

RANDY HULTGREN, Illinois 

JOHN R. MOOLENAAR, Michigan 

BRUCE WESTERMAN, Arkansas 

DAN NEWHOUSE, Washington 

GARY PALMER, Alabama 

RALPH LEE ABRAHAM, Louisiana 

LAMAR S. SMITH, Texas 

DANIEL LIPINSKI, Illinois 

ELIZABETH H. ESTY, Connecticut 

KATHERINE M. CLARK, Massachusetts 

PAUL TONKO, New York 

SUZANNE BONAMICI, Oregon 

ERIC SWALWELL, California 

EDDIE BERNICE JOHNSON, Texas 
(III) 

C O N T E N T S 

June 16, 2015 

Page 

Witness List ............................................................................................................. 2 

Hearing Charter ...................................................................................................... 3 

Opening Statements 

Statement by Representative Barbara Comstock, Chairwoman, Subcommittee 

on Research, Committee on Science, Space, and Technology, U.S. House 

of Representatives ................................................................................................ 5 

Written Statement ............................................................................................ 5 

Statement by Representative Daniel Lipinski, Ranking Minority Member, 

Subcommittee on Research, Committee on Science, Space, and Technology, 

U.S. House of Representatives ............................................................................ 6 

Written Statement ............................................................................................ 7 

Statement by Representative Lamar S. Smith, Chairman, Committee on 

Science, Space, and Technology, U.S. House of Representatives ..................... 8 

Written Statement ............................................................................................ 9 

Witnesses: 

Dr. Victor J. Dzau, President, Institute of Medicine, the National Academy 

of Sciences 

Oral Statement ................................................................................................. 10 

Written Statement ............................................................................................ 13 

Dr. Jennifer Doudna, Professor of Biochemistry and Molecular Biology, University of California, 

Oral Statement ................................................................................................. 18 

Written Statement ............................................................................................ 20 

Dr. Elizabeth McNally, Professor of Genetic Medicine, Professor in MedicineCardiology and Biochemistry and Molecular Genetics; Director, Center for 

Genetic Medicine, Northwestern University 

Oral Statement ................................................................................................. 23 

Written Statement ............................................................................................ 25 

Dr. Jeffrey Kahn, Professor of Bioethics and Public Policy; Deputy Director 

for Policy and Administration, Berman Institute of Bioethics, Johns Hopkins University 

Oral Statement ................................................................................................. 32 

Written Statement ............................................................................................ 34 

Discussion ................................................................................................................. 40 

Appendix I: Answers to Post-Hearing Questions 

Dr. Elizabeth McNally, Professor of Genetic Medicine, Professor in MedicineCardiology and Biochemistry and Molecular Genetics; Director, Center for 

Genetic Medicine, Northwestern University ...................................................... 64 

Appendix II: Additional Material for the Record 

Statement by Representative Eddie Bernice Johnson, Ranking Member, Committee on Science, Space, and Technology, U.S. House of Representatives .... 66 

(1) 

THE SCIENCE AND ETHICS OF 

GENETICALLY ENGINEERED HUMAN DNA 

TUESDAY, JUNE 16, 2015 

HOUSE OF REPRESENTATIVES, 

SUBCOMMITTEE ON RESEARCH AND TECHNOLOGY

COMMITTEE ON SCIENCE, SPACE, AND TECHNOLOGY, 

Washington, D.C. 

The Subcommittee met, pursuant to call, at 2:16 p.m., in Room 

2318 of the Rayburn House Office Building, Hon. Barbara Comstock [Chairwoman of the Subcommittee] presiding. 
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Chairwoman COMSTOCK. The Subcommittee on Research and 

Technology will come to order. Without objection, the Chair is authorized to declare recesses of the Subcommittee at any time. 

And without objection, the gentleman from California, Mr. Sherman, is authorized to participate in today’s hearing. 

Good afternoon, and welcome to this hearing entitled ‘‘The 

Science and Ethics of Genetically Engineered Human DNA.’’ 

And I believe we also would like to welcome Representative 

Abraham to his first Science Committee hearing. Dr. Abraham, we 

are happy to have you join the Research and Technology Subcommittee and we look forward to having the benefit of your expertise. 

Now, in front of you are packets containing the written testimonies, biographies, and truth-in-testimony disclosures for today’s 

witnesses. 

I now recognize myself for an opening statement. 

Biotechnology—the engineering of genetic material in living 

beings and plants—has transformed modern medicine and agriculture. Rapid advances in biotech research have brought great opportunities for new medical treatments and products, and simultaneously have also raised questions about possible ethical implications and safety issues. 

Today, we are here to discuss the science and ethics of the most 

recent and eye-opening development in biotechnology: human genome-editing. This research has been a major topic of news and 

editorials in recent months. New tools that allow a gene to be deleted, inserted, or replaced by a different piece of DNA are becoming more cost-effective and simpler to execute. 

In April it was reported that for the first time a team of Chinese 

scientists had attempted to edit the genome of human embryos. 

The report raised concerns for many scientists and policymakers 

about the safety and ethics of using these new technologies on 

human DNA. Many prominent scientists have called for a better 

framework to be developed for responsible use of the technology. 

I look forward to learning more from our witnesses today who 

will provide an overview of the science behind these new technologies, help us examine the implications and risks, and explore 

what the next steps should be for building the right kind of framework for utilizing the technology. They will also help us answer 

how the United States can be a leader and provide scientific and 

ethical leadership in this arena. 

[The prepared statement of Chairwoman Comstock follows:] 

PREPARED STATEMENT OF SUBCOMMITTEE

CHAIRWOMAN BARBARA COMSTOCK

Biotechnology—the engineering of genetic material in living beings and plants— 

has transformed modern medicine and agriculture. 

Rapid advances in biotech research have brought great opportunities for new medical treatments and products, and simultaneously have also raised questions about 

possible ethical implications and safety issues. 

Today, we are here to discuss the science and ethics of the most recent and eyeopening development in biotechnology: human genome-editing. 

This research has been a major topic of news and editorials in recent months. 

New tools that allow a gene to be deleted, inserted, or replaced by a different piece 

of DNA are becoming more cost-effective and simpler to execute. 
6 

In April, it was reported that for the first time a team of Chinese scientists had 

attempted to edit the genome of human embryos. The report raised concerns for 

many scientists and policy makers about the safety and ethics of using these new 

technologies on human DNA. 

Many prominent scientists have called for a better framework to be developed for 

responsible use of the technology. 

I look forward to learning more from our witnesses today who will provide an 

overview of the science behind these new technologies, help us examine the ethical 

implications and risks, and explore what the next steps should be for building a responsible framework for utilizing the technology. They will also help us answer how 

the United States can provide scientific and ethical leadership in this arena. 

Chairwoman COMSTOCK. So I now recognize the Ranking Member, the gentleman from Illinois, for his opening statement. 

Mr. LIPINSKI. Thank you, Chairwoman Comstock, for holding this 

hearing on the science and ethics of new gene editing technologies. 

I want to thank all the witnesses for being here today and look 

forward to your testimony. 

Although we’re talking about gene editing technologies that are 

very new, it’s important to mention that humans have been altering the genomes of species through selective breeding for thousands 

of years. And since the 1970s, it has been possible to directly manipulate DNA, which led to a biotechnology revolution and significant economic growth. 

Then we had the Human Genome Project to sequence the human 

genome, and it was coordinated by the Department of Energy and 

the National Institutes of Health. The full human genome was 

sequenced in 2003, opening up whole new possibilities for diagnosing and treating diseases. One such pathway led to the invention of the CRISPR technology. 

Thanks to new gene editing technologies, which include CRISPR, 

we’re able to add, remove, and replace DNA bases. They can be 

thought of as search-and-replace tools for DNA. They’re incredibly 

powerful technologies that have the potential to transform the 

healthcare, energy, and agricultural sectors. Although new, these 

technologies were the outgrowth of decades of fundamental research, some of which was supported by the National Science Foundation. 

We are here today because a Chinese research group recently 

published a paper in which they used these technologies to try to 

modify human embryos. That paper highlights scientific and ethical issues with these technologies, especially if they are being used 

to modify human germline cells as opposed to adult somatic cells. 

I look forward to hearing about the science behind these technologies, as well as how the United States can be a leader in addressing the safety and ethical concerns associated with them. 

I understand the National Academies has launched a major initiative around human gene editing technologies. In the 1970s, the 

National Academies played a similar role dealing with the thennew biotechnologies, and I look forward to hearing more about 

what they’re planning to do concerning these new gene editing 

technologies. I also look forward to hearing about some of the potential nonhuman applications. 

[The prepared statement of Mr. Lipinski follows:] 
7 

PREPARED STATEMENT OF SUBCOMMITTEE

MINORITY RANKING MEMBER DANIEL LIPINSKI

Thank you Chairwoman Comstock for holding this hearing on the science and ethics of new gene editing technologies. I want to thank all the witnesses for being here 

this afternoon and I look forward to hearing your testimony. 

Although we are talking about gene editing technologies that are very new, it is 

important to mention that humans have been altering the genomes of species 

through selective breeding for thousands of years. Since the 1970s, it has been possible to directly manipulate DNA, which led to a biotechnology revolution and significant economic growth. Then we had the Human Genome Project to sequence the 

human genome that was coordinated by the Department of Energy and the National 

Institutes of Health. The full human genome was sequenced in 2003, opening up 

whole new possibilities for diagnosing and treating diseases. One such pathway led 

to the invention of the CRISPR technology. 

Thanks to new gene editing technologies, which include CRISPR, we are able to 

add, remove, and replace DNA bases. They can be thought of as ‘‘search and replace’’ tools for DNA. They are incredibly powerful technologies that have the potential to transform the health care, energy, and agricultural sectors. Although new, 

these technologies were the outgrowth of decades of fundamental research, some of 

which was supported by the National Science Foundation. We are here today because a Chinese research group recently published a paper in which they used these 

technologies to try to modify human embryos. That paper highlights scientific and 

ethical issues with these technologies, especially if they are being used to modify 

human germline cells as opposed to adult somatic cells. 

I look forward to hearing about the science behind these technologies as well as 

how the United States can be a leader in addressing the safety and ethical concerns 

associated with them. I understand that the National Academies has launched a 

major initiative around human gene editing technologies. In the 1970s, the National 

Academies played a similar role dealing with the then-new biotechnologies and I 

look forward to hearing more about what they are planning to do concerning these 

new gene editing technologies. I also look forward to hearing about some of the potential non-human applications. 

Now I would like to yield my remaining time to my colleague from Illinois, Mr. 

Foster, who is very interested in this topic and helped organize today’s hearing. 

Mr. LIPINSKI. With that, I’d like to yield my remaining time to 

my colleague and neighbor from Illinois, Dr. Foster, who was very 

interested in this topic and helped to organize today’s hearing. 

Mr. FOSTER. Thank you, and I’d like to thank the whole Research 

and Technology Subcommittee, including the Chair, Congresswoman Comstock, and Ranking Member Lipinski for allowing me 

to join you here today. And similarly, I wanted to thank Chairman 

Smith and Ranking Member Johnson for agreeing to hold this 

hearing. And a very special thank you to the witnesses for taking 

their time out for this very important issue. 

It is rare that prominent members of the scientific community 

come together to warn our leaders of technological breakthroughs 

that our legal system and society may not be prepared for, and yet, 

this is exactly what appears to be happening with recent discoveries in genetic editing tools. 

As the last Ph.D. scientist in Congress, I am afraid I’ve served 

a sort of a lightning rod for many of these warnings and I take 

them very seriously. 

I want to commend the National Academy of Sciences and the Institute of Medicine for the launch of their major initiative on 

human gene editing, and I want to make sure that Congress does 

everything constructive that it can to make sure that this is handled responsibly. 

There is the possibility of very great benefits from these new 

technologies, and what makes them really revolutionary is what 
8 

they can mean for humans, for example, replacing bone marrow of 

someone suffering from sickle cell disease with a modified version 

of their own marrow with the genetic defect removed. 

However, if genetic modifications are made to so-called germline 

cells—these are sperm, eggs, embryos—then the modifications will 

be carried forward to future generations, which has implications 

that we need to carefully consider. We’re on the verge of a technological breakthrough that could change the future of mankind and 

we must not blindly charge ahead. 

Thank you, and I yield back my time. 

Mr. LIPINSKI. I will just conclude. I agree with Dr. Foster and it’s 

great to see that we have so many people here at this hearing. And 

it’s a very important issue that we really need to consider deeply, 

so I thank the Chairwoman and the Chairman of the Full Committee, Chairman Smith, for holding this hearing today, and I’ll 

yield back. 

Chairwoman COMSTOCK. Thank you. 

And I now recognize the Chairman of the Full Committee, Mr. 

Smith. 

Chairman SMITH. Thank you, Madam Chair. 

I do look forward, as do the others, to today’s discussion on a new 

development in biology, which has been called ‘‘a game changer,’’ 

‘‘revolutionary,’’ ‘‘powerful,’’ and ‘‘a major issue for all humanity.’’ 

The new discoveries in genetically engineering human DNA offer 

potential cures for devastating genetic disorders. But the speed at 

which these new, simpler, and cheaper technologies are being used 

in the lab also presents ethical and health concerns. Most of the 

scientific community members have been clear: the science and ethics of this new technology must be resolved in order to prevent dangerous abuses and unintended consequences. 

A recent report from China, where teams of researchers have 

begun to experiment with engineering DNA in human embryos, is 

alarming. This is an area where the United States can and should 

provide scientific and moral leadership. We need to better understand the technology and procedures being used so that we can ensure patients are treated in the safest and most ethical manner 

possible. 

An April editorial in Science magazine called for a prudent path 

forward for genomic engineering. It recommended a moratorium on 

further research, while creating public forums for scientists, 

ethicists, and policymakers to discover the—to discuss ‘‘the attendant ethical, social, and legal implications of genome modification.’’ 

This is why it is important that the House Science Committee is 

holding the first Congressional hearing on this profound and complex subject. 

The purpose of the Science Committee is to explore the significance of scientific discoveries, as well as their potential implications for humankind. But we also must always be conscious of the 

potential ethical and moral issues raised by previously unimagined 

scientific breakthroughs. We must take the lead in reviewing new 

and innovative areas of science, such as genetically engineered 

DNA. 
9 

So I look forward, Madam Chair, to this informative discussion 

today, and we have an excellent panel of witnesses to hear from 

as well. 

And I’ll yield back. 

[The prepared statement of Chairman Smith follows:] 

PREPARED STATEMENT OF COMMITTEE ON SCIENCE, SPACE, AND TECHNOLOGY

CHAIRMAN LAMAR SMITH

Thank you Madam Chair. I look forward to today’s discussion on a new development in biology, which has been called ‘‘a game changer,’’ ‘‘revolutionary,’’ ‘‘powerful,’’ and ‘‘a major issue for all humanity.’’ 

The new discoveries in genetically engineering human DNA offer potential cures 

for devastating genetic disorders. But the speed at which these new, simpler and 

cheaper technologies are being used in the lab also presents ethical and health concerns. Most of the scientific community members have been clear: the science and ethics 

of this new technology must be resolved in order to prevent dangerous abuses and 

unintended consequences. 

A recent report from China, where teams of researchers have begun to experiment 

with engineering DNA in human embryos, is alarming. This is an area where the 

United States can and should provide scientific and moral leadership. 

We need to better understand the technology and procedures being used so that 

we can ensure patients are treated in the safest and most ethical manner possible. 

An April editorial in Science Magazine called for a prudent path forward for 

genomic engineering. It recommended a moratorium on further research, while creating public forums for scientists, ethicists and policy makers to discuss ‘‘the attendant ethical, social, and legal implications of genome modification.’’ 

This is why it is important that the House Science Committee is holding the first 

congressional hearing on this profound and complex subject. 

The purpose of the Science Committee is to explore the significance of scientific 

discoveries as well as their potential implications for humankind. 

But we also must always be conscious of the potential ethical and moral issues 

raised by previously unimagined scientific breakthroughs. 

We must take the lead in reviewing new and innovative areas of science, such as 

genetically engineered DNA. 

I look forward to an informative discussion with our distinguished panel of wit- nesses. 

Chairwoman COMSTOCK. Thank you. 

And if there are Members who wish to submit additional opening 

statements, your statements will be added to the record at this 

point. 

Now, at this time I would like to introduce our witnesses. Dr. 

Victor Dzau is the President of the National Academies Institute 

of Medicine and the James B. Duke Professor of Medicine at Duke 

University. Dr. Dzau has received many honors, including the Distinguished Scientist Award from the American Heart Association. 

He earned his undergraduate and medical degrees from McGill 

University and holds eight honorary doctorates. 

Our second witness today is Dr. Jennifer Doudna. I think I got 

that. Dr. Doudna is Professor of Molecular and Cell Biology and 

Professor of Chemistry at U.C. Berkeley. A member of the National 

Academy of Sciences, Dr. Doudna is the recipient of several 

awards, including the NSF Waterman Award and the 2015 Breakthrough Prize for Life Sciences. Dr. Doudna earned her undergraduate degree in biochemistry from Pomona College and her 

Ph.D. in biological chemistry from Harvard University. 

I now recognize the gentleman from Illinois, Mr. Lipinski, to introduce our next witness. 

Mr. LIPINSKI. Thank you. 
10 

As a Northwestern University alumnus, I’m very excited to have 

Dr. McNally here today. To say an aside, Dr. Dzau, I’m also an 

alum of Duke University, and unfortunately for Dr. Doudna, an 

alum of Stanford also. 

Dr. McNally is the Director of the Center for Genetic Medicine 

and Professor in the Departments of Medicine and Biochemistry at 

Northwestern University’s Feinberg School of Medicine. She is a 

cardiologist who specializes in inherited forms of heart disease. Dr. 

McNally’s research has identified genes and mechanisms for how 

genetic lead to heart and muscle disease. She has an undergraduate degree in biology and philosophy from Barnard College at 

Columbia University and an M.D. and Ph.D. from the Albert Einstein College of Medicine. 

It is my pleasure to welcome Dr. McNally to our committee and 

look forward to her testimony. 

Chairwoman COMSTOCK. Okay. And our final witness is Dr. Jeffrey Kahn, the Robert Henry Levi and Ryda Hecht Levi Professor 

of Bioethics and Public Policy at the Johns Hopkins Berman Institute of Bioethics and a Professor in the Department of Health Policy and Management at the Johns Hopkins Bloomberg School of 

Public Health. Dr. Khan received his bachelor’s in microbiology 

from the University of California, Los Angeles, his master’s in public health from Johns Hopkins, and his Ph.D. in philosophy and 

bioethics from Georgetown University. 

In order to allow time for discussion, we would ask that you limit 

your testimony to five minutes and your entire written statement 

will be made part of the record. 

I now recognize Dr. Dzau for five minutes to present his testimony. 

TESTIMONY OF DR. VICTOR J. DZAU, PRESIDENT, 

INSTITUTE OF MEDICINE, 

THE NATIONAL ACADEMY OF SCIENCES 

Dr. DZAU. Good afternoon, Chairman Smith, Chairwoman Comstock, Ranking Member Lipinski, and Subcommittee Members. As 

you heard, I’m Victor Dzau. I’m the President of the Institute of 

Medicine, which will soon be named the National Academy of Medicine on July 1. 

I’m pleased to be here on behalf of the National Academies of 

Sciences, Engineering, and Medicine. The Academies operate under 

a Congressional Charter signed by Abraham Lincoln to provide advice to the Nation on matters where science, technology, and medicine can solve complex challenges and thereby improve people’s 

lives. 

Thank you for the opportunity to speak with you today about this 

very important matter of human gene editing and the major initiative that we have at the National Academies launched to help 

guide decision-making in this area. 

The Academies have an established record of leadership on advising on emerging and often controversial areas of science, particularly genetic research, such as recombinant DNA and stem cell research. Our initiative is marshaling the best scientific evidence, 

medical, ethical, legal, and other expertise to help you and the Nation obtain a thorough understanding of gene editing and its poten-
11 

tial risks and benefits. Our work is intended to provide a solid 

foundation to help inform decisions and policies on this research. 

As you will hear from other witnesses today, gene editing technology holds great promise. In fact, powerful new tools such as 

CRISPR/Cas9 developed by my colleague Dr. Doudna and others, 

as well as other genetic engineering technology, allow researchers 

now to precisely modify the genetic makeup of any living organism, 

including humans. The possible benefit application for such technologies are many. They could offer a cure to devastating genetic 

diseases such as Huntington’s disease, as you heard, or sickle cell 

anemia. It can help improve and understand the treatment of 

many other illnesses. 

These technologies also present complex challenges both to scientific and medical communities and to society as a whole. Of particular concern is the potential to make permanent modification to 

human DNA in nuclei of eggs, sperm, or human embryos that are 

then passed down to succeeding generations known as germline 

gene editing. Research that attempts to alter the human germline 

raises important issues in so many different ways about safety, 

risk, social, economic, ethical, and regulatory considerations. So although more remains to be done before these technologies can be 

deployed safely, their availability certainly intensifies this debate 

among scientists and physicians about such research. 

Here in the United States there are legislative prohibitions on 

the use of federal funds for research of human embryos and there 

exist constraints on such research when subject to oversight by the 

U.S. Food and Drug Administration or other government agencies. 

These constraints, however, do not apply to work done internationally without federal funds and without the intent to seek federal 

approval of any product of that research. So clearly, we have 

reached a critical juncture in genetic editing research and guidance 

is needed. 

The National Academies of Sciences, Engineering, and Medicine 

are prepared to provide that guidance based on an in-depth review 

of science underlying gene editing, the potential benefits, and the 

valid concerns raised by this research. Toward that end, our initiative on human gene editing research is already underway. Just last 

week, we held the first meeting of a multidisciplinary advisory 

group that will help us steer our initiative and ensure the Academies’ efforts are comprehensive, inclusive, and transparent. 

Since much of this research is done internationally, the Academies will convene a global summit to obtain multinational perspectives on recent scientific development in human gene editing 

and the associated ethical and governance issues. Concurrently, 

we’ll appoint an expert committee to conduct a comprehensive 

study of the scientific underpinning and clinical, ethical, legal, and 

social implications of human gene editing. We hope that we will 

come up with recommendations which can inform the Nation for 

decisions in this area. 

All of us—scientists, physicians, policymakers, and public—want 

to do everything possible to ensure the scientific and medical 

breakthroughs benefit all of mankind and do no harm. The Academies are certainly ready to help. 
12 

I would be very happy to answer any questions the Subcommittee may have. Thank you. 

[The prepared statement of Dr. Dzau follows:] 
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Chairwoman COMSTOCK. And Dr. Doudna. 

TESTIMONY OF DR. JENNIFER DOUDNA, 

PROFESSOR OF BIOCHEMISTRY AND MOLECULAR BIOLOGY, 

UNIVERSITY OF CALIFORNIA, BERKELEY 

Dr. DOUDNA. Good afternoon, Chairwoman Comstock and the 

rest of the Members of the Committee. It’s a great pleasure for me 

to be here and have the opportunity to talk with you about science 

that I’ve been involved with from its origin and involved in leading 

the discussion of where it’s going. 

I wanted to start by saying that this is research that originated 

as a basic science project funded in part by the National Science 

Foundation. We did not aim to develop a genome editing technology 

but in the course of the experiments that we were doing, it became 

clear that what started as a study of a bacterial immune system, 

the way bacteria fight the flu, could actually be reengineered and 

re-harnessed really as a technology for changing sequences in the 

genomes of cells and whole organisms. 

I wanted to tell you a little bit more about the science behind 

this to explain a little bit about how it works and why it’s revolutionary. So I think what really makes this distinct from other ways 

of manipulating DNA and cells is that it’s a very simple system. 

It relies I would really make the analogy to software that you use 

for your computer. Here we have a protein called Cas9 that can be 

easily reprogrammed by using a short piece of nucleic acid called 

RNA that enables this protein to be directed to essentially any 

DNA sequence in a genome of an organism. And because genome 

sequencing has become very prevalent and is becoming less and 

less expensive, we have an exciting convergence of technologies 

that give us information about the entire genome in a cell or an 

organism and now a tool that allows scientists to change that sequence in a very precise fashion so we can do things, as was mentioned in the opening statements, like correct mutations that would 

otherwise lead to genetic disease. 

So this is, I think, a very exciting moment in biology. It’s opened 

up a lot of opportunities for research, for clinical applications in the 

future, but it also raises various questions about the way that this 

technology should be employed going forward, and in particular in 

our discussion today the question of whether and when this technology should be employed to change the sequence in the human 

germline in eggs or sperm or embryos that would lead to a genetically modified person that would be a mutation that could be 

passed down to their children. 

And I realized fairly early on in our research that this technology 

was likely to be applicable in the human germline, and that led me 

to initiate a discussion initially with some—a fairly small group of 

scientists in California. We met in January of this year in the 

Napa Valley to discuss this very issue and we spent a day. We 

had—that small meeting included scientists, clinicians, as well as 

bioethicists to discuss the various issues around human germline 

editing, and that meeting resulted in a perspective that was published in Science magazine about two months ago that was referred 

to in the opening statements for—that called for a prudent path 
19 

forward in any kind of clinical application of germline editing in 

humans. 

I do want to point out that our perspective favors research in this 

area. I think we feel as scientists that it’s very important to have 

data so that we can make informed decisions about future potential 

applications, and I think this is a—I think many of us appreciate 

this is a technology that could be very helpful for people that have 

inherited genetic disorders. However, to ensure that any kind of 

application clinically in the human germline was safe and really 

was used in an ethical fashion, we do need to understand how this 

technology operates in those types of cells. 

Thank you. 

[The prepared statement of Dr. Doudna follows:] 
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Chairwoman COMSTOCK. Thank you. 

Now, we’ll hear from Dr. McNally. 

TESTIMONY OF DR. ELIZABETH MCNALLY, 

PROFESSOR OF GENETIC MEDICINE, 

PROFESSOR IN MEDICINE-CARDIOLOGY AND BIOCHEMISTRY 

AND MOLECULAR GENETICS; 

DIRECTOR, CENTER FOR GENETIC MEDICINE, 

NORTHWESTERN UNIVERSITY 

Dr. MCNALLY. Thank you. 

On behalf of Northwestern University, I’d like to thank Chairwoman Comstock and Ranking Member Lipinski for inviting me 

here today. 

I’m Elizabeth McNally. I’m the Ward Professor of Genetic Medicine, and I direct the Center for Genetic Medicine at Northwestern. 

I’m a cardiologist and I specialize in providing care for patients and 

families with inherited forms of heart disease. Over the last decade, we’ve seen a dramatic increase in available genetic testing and 

we now routinely provide genetic diagnosis, risk assessment, and 

importantly, risk reduction for genetic diseases that affect the 

heart. 

Diseases like cystic fibrosis, Duchenne muscular dystrophy, sickle cell anemia are those that are caused by mutations in single 

genes. The gene editing techniques that we are here discussing 

offer the opportunity for permanent lifelong treatment of those disorders. With advances in DNA sequencing technology, it is now 

possible to sequence an individual genome in a day. For less than 

the cost of an MRI, a genome can be analyzed with high accuracy 

pinpointing single gene mutations. The Office of Rare Diseases 

identifies nearly 7,000 rare diseases and many of these are genetic 

in origin, often arising from single mutations. The ORD estimates 

that nearly 30 million Americans are affected by rare diseases. 

More than half of rare diseases affect children. 

Concomitant with advances in genetic diagnoses, there are parallel leaps in genome editing. CRISPR/Cas9 represents a significant advance for genome editing. Because of the co-development of 

gene editing and stem cell biology, there is significant potential 

clinical application. Induced pluripotent human stem cells can be 

made from blood, skin, and other mature human cells. For my 

field, cardiology, skin cells can actually form beating heart-like 

cells in a dish allowing us to discern how mutations cause disease 

and letting us test how to correct these diseases. The human population is not placed at risk by these experiments in cells and it 

seems fair to say that the human population would actually be 

harmed by not doing these experiments since the research offers 

the potent opportunity to improve human health. 

Stem cells of the bone marrow, muscle, skin, and other organs 

can be isolated and edited. With these methods, it would be possible to cure sickle cell anemia or Duchenne muscular dystrophy. 

In mice, CRISPR/Cas9 mediated correction of fertilized eggs corrected the defect for Duchenne muscular dystrophy. The method, 

while imperfect, was associated with a remarkably high correction 

rate. 
24 

Recently, a group of distinguished scientists called for careful 

consideration of gene editing in fertilized oocytes fearing the potential for germline gene editing and ultimately human eugenics. 

These discussions were enhanced and prompted by the recent report which we’ve heard about from Liang, et al., which described 

the efforts using CRISPR/Cas9 in fertilized oocytes. 

A regulatory framework for gene editing should encompass several key points. It should permit research to optimize and improve 

CRISPR/Cas9 and related technologies. It should permit in vitro 

and cell-based gene editing technologies, including those in embryonic and induced pluripotent stem cells. It should permit in vitro 

and cell-based editing with the intent to reintroduce cells into humans as a therapeutic measure for somatic cells. And it should permit the generation of gene-edited animals for the purposes of scientific research. It may consider limiting or even prohibiting gene 

editing under the circumstances where human transmission of 

gene-edited germ lines would occur. 

But would we ever really consider germline gene editing? So we 

should consider the scenario of pre-implantation genetic diagnosis, 

otherwise known as PGD. PGD is pursued by families to avoid 

transmitting genetic diseases. PGD involves in vitro fertilization 

coupled with genetic testing. PGD is not covered by insurance, and 

yet for some families, they make this choice. These may be families 

who are already struggling with caring for one disabled child and 

who cannot care for a second disabled child. 

PGD is a personal option and one that is made solely by parents 

and families. In principle, it is possible that the efficiency of genome editing will improve so that pre-implantation genetic correction could accompany PGD. With this process, gene editing to correct and eliminate a genetic disease could become reality. While 

the temptation may be to ban or limit this possibility, we should 

do so only with caution. Parents of children with genetic disease 

express significant concern, responsibility, and often dismay for 

having passed on mutations to their children. A parent’s desire to 

protect children is undeniable. As a society and as a nation, we 

protect children. 

It may be tempting and easiest to ban gene editing where 

germline transmission could occur, yet the justified use of this approach is certainly conceivable and may one day be appropriate. 

Thank you. 

[The prepared statement of Dr. McNally follows:] 
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Chairwoman COMSTOCK. Thank you. 

And Dr. Khan. 

TESTIMONY OF DR. JEFFREY KAHN, 

PROFESSOR OF BIOETHICS AND PUBLIC POLICY; 

DEPUTY DIRECTOR FOR POLICY AND ADMINISTRATION, 

BERMAN INSTITUTE OF BIOETHICS, 

JOHNS HOPKINS UNIVERSITY 

Dr. KAHN. Thank you. Chairwoman Comstock, Chairman Smith, 

and Ranking Member Lipinski, thank you for the opportunity to 

testify today on this timely and vitally important subject. 

As you heard in the introductions, I am a Professor of Bioethics 

in Public Policy at the Johns Hopkins Berman Institute of Bioethics in Baltimore. 

Also relevant to my comments today, I am also currently Chair 

of an Institute of Medicine Consensus Study commissioned by the 

FDA on ethical and social policy considerations of novel techniques 

for prevention of mitochondrial transmission in women to their offspring. Given that this study is considering issues related to the 

topic of today’s hearing and the work of that committee is ongoing, 

I will restrict my comments to general observations and an overview of ethical and policy landscape related to gene editing. 

I’ll focus my comments on three main topics: first, policy history 

and related areas of science and biomedical research; second, ethical issues raised by gene editing technologies; and third, relevant 

existing ethical frameworks and approaches to oversight. 

The relevant policy history started in 1975, and we heard some 

mention of this earlier, with the Asilomar Conference on recombinant DNA molecules whose summary statement focused on containment of the risks of creating and working with genetically 

modified organisms. And with the admonition to avoid experiments 

that ‘‘pose such serious dangers that their performance should not 

be undertaken at this time,’’ along with a call for continuing reassessment of issues arising in light of new knowledge gained with 

the experience of the then-new genetic technologies. And we’ve 

heard from Dr. Dzau that the National Academies are effectively 

taking on the same role 40 years later. 

These voluntary suggestions that came from the Asilomar summary gave way to more robust oversight as the use of genetic technologies became more refined and with the initial attempts to treat 

disease in humans. It should be noted that that’s 40 years ago, and 

in the current environment, it would be very difficult for voluntary 

statements from a collection of even esteemed U.S. scientists to 

prevent research from going forward internationally, as we’ve seen 

already with the publication of the Chinese laboratory experiment. 

The ethical issues posed by gene editing and related technologies 

for modifying human DNA fall into three general categories of concern: first, the implications of the modification of germline DNA, 

and we’ve heard some about that already; second, the implications 

of interfering in processes that should be off-limits to humans. 

Sometimes those are sort of generally termed ‘‘playing God,’’ and 

that’s problematic in some people’s minds; and third, the potential 

for selection or introduction of traits for other than treatment or 
33 

avoidance of disease, such as physical or behavioral traits or even 

enhancements. 

The focus of much ethical analysis in the application of manipulation of genetic information in humans has been on changes that 

affect the germline, that is changes that are heritable and therefore 

able to be passed on to future generations of individuals. The basis 

of these concerns relate to the uncertainty of the effects of genetic 

notification, the inability to ‘‘undo’’ unintended genetic changes or 

limit their effects, and the risks of passing on such unintended 

changes and their consequences to future generations. And that 

would of course go on forever. 

These ethical concerns have been addressed through a range of 

approaches in order to limit certain types of research or to provide 

prospective oversight prior to particular proposals being undertaken. We heard from Dr. Dzau that there are restrictions on federal funding of research that involves human embryos. Privately 

funded research is not affected by these restrictions, though the 

convention is that research on embryos should take place no later 

than 14 days after fertilization, and that’s a limit also accepted by 

most countries engaged in research on human embryos. I should 

also note that there seems to be growing agreement that research 

should be restricted to nonviable human embryos. 

There are a number of institution-level oversight mechanisms 

that will apply to gene editing. That includes Institutional Biosafety Committees, which are charged with overseeing research 

with recombinant or synthetic nucleic acid molecules; Institutional 

Stem Cell Research Oversight Committees, which are charged 

with, as their name implies, research on human embryonic stem 

cells and related areas of research. And as the technologies are introduced into human subjects, Institutional Review Boards will be 

charged with overseeing any potential use on humans. 

Lastly, there’s a role to be played by the scientific publication 

community. They have—journal publishers have an increasingly 

important role to play in setting and enforcing standards of behavior within the scientific community since publication of findings in 

the peer-reviewed scientific literature signifies the endorsement of 

the community of researchers. 

Journals also play an additional critical role with requirements 

on the ethics standards being respected, assurances authors’ contributions are duly noted, and that human subjects are protected. 

So there’s a role to be played on the part of the journal publication 

process that will restrict the potential of any unethical research 

going forward. 

Let me conclude by saying the United States has played a leadership role in this area in recombinant DNA and has the opportunity 

to do so going forward. There will be gaps identified in the process 

that the National Academies has set out on that need to be identified and addressed, and it is an appropriate time to consider what 

those ought to be. 

Thank you. 

[The prepared statement of Dr. Kahn follows:] 
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Chairwoman COMSTOCK. I thank the witnesses for their very interesting testimony. 

And now I remind Members that Committee rules limit questioning for five minutes. 

And the Chair—as Chair, I recognize myself for five minutes. 

So I wanted to hear a little bit more, Dr. Doudna, from what you 

mentioned where you had initiated the discussion with the scientists in California and bioethicists and others to address this. 

Some of the key—and maybe expand a little bit more on some of 

the key issues you found and how we avoid maybe where China 

might go and how we do define those boundaries and kind of what 

some of the concerns were that were raised. And if anyone else 

would like to address that, too, but I thought I’d start with you and 

maybe expounding a little bit more on this group. 

Dr. DOUDNA. Sure, thank you. 

I think, you know, what was interesting to me in that conversation was that it was a wide-ranging discussion that started with 

sort of the—maybe the way that we’ve been discussing this technology so far here in the Committee and eventually got to a point 

where, as somebody around the table said, you know, there may 

come a time when we would consider it unethical not to do editing 

in the germline for certain kinds of applications such as some of 

the things that Dr. McNally mentioned. 

So I think that it’s very important to appreciate that, you know, 

this is a powerful technology that is—you know, we’re sort of looking at it here from the perspective of safety and ethical concerns 

but I think they’re also—you know, that can be turned around. And 

that was something that came out of our discussion in California 

that I found very interesting. 

And the other thing that we discussed was the fact that, you 

know, this technology, unlike previous technologies for genome editing, is very simple to employ relatively. I mean it’s something 

that, you know, people that have expertise in molecular biology can 

fairly readily use in their laboratories. So I think, you know, the 

reality is that it would be very hard to really put regulations on 

this in terms of research applications. 

And I think that means that we just have to be thoughtful about 

providing leadership in terms of the—you know, the—as Dr. Kahn 

was saying, in terms of the way papers are published and reviewed 

among scientists so that, you know, the scientific community helps 

to provide the kind of direction and, you know, vision for the way 

this should move forward that hopefully will be respected by many 

others. But I think the reality is that it—you know, it is a technology that’s just very widely available now and is being employed 

worldwide. 

Dr. DZAU. I’d like to echo what Dr. Doudna said. I think we at 

the Academies feel very privileged to be asked by the scientists— 

Ralph Cicerone, the President of National Academy of Science, myself, got lots of phone calls and emails to say, you know, the Academy really should take this on because we have done so in the 

past, as you heard, during the time of recombinant DNA, Asilomar 

conference, the human cloning, and also the whole issue of stem 

cell guidelines, as well as mapping the human genome. So we have 

to do this. 
41 

I think that in the context of what’s being discussed, it’s really 

important that we have people from all different disciplines, not 

only scientists but ethicists, legal experts, medical physicians, and 

others all engage in this discussion because at the end of the day 

what we want to do is to find what appears to be the consensus 

of what the right thing to do is for our country and so we look forward to working on this. 

Dr. KAHN. So I would just like to echo what you’ve heard from 

my colleagues on the panel and add a little bit to expand on what 

I said about publication. It’s difficult for the scientific community 

to identify any one thing that it can take on in an international 

way, but scientific publication knows no borders. And there have 

been calls now for—before publication of any gene editing research 

that there be a disclosure of the ethics review that that research 

underwent. That did not happen so far as we know with the Chinese research that was recently published, and in fact, the reports 

have been about Science and Nature rejected that paper when they 

considered it and at least in part on ethics grounds. So that would 

be one way to create some—a framing of what would be acceptable 

as determined by the scientific community itself through the peer 

review process. 

Chairwoman COMSTOCK. Thank you. And, Dr. McNally, did you 

have anything to add? I have a few more seconds before I turn—— 

Dr. MCNALLY. I would also remember that through this process 

it’s not just the scientists and the physicians and the regulatory 

bodies but to also remember the patient advocacy groups and the 

patients themselves. They’re going to be the loudest voice in this 

process and we need to hear what they have to say. 

Chairwoman COMSTOCK. Thank you. I appreciate it. 

Now I recognize Mr. Lipinski for five minutes. 

Mr. LIPINSKI. Thank you. 

Before I follow up, I just want to make sure we’re more clear on 

this when we get into the subject. 

Dr. Doudna, you and the group that have met, you’ve called for 

a temporary moratorium on the use of the technologies on human 

embryos. Is that correct? And what led you to that and are there 

particular milestones and discussions of the science that you’re 

working towards and then see ending the moratorium? And I want 

to get the other panelists to comment on that. 

Dr. DOUDNA. So I would say what led us to that initial meeting 

in California was the appreciation that this technology would likely, you know, be functional in the human germline, and furthermore, that it was possible that people could do this fairly easily 

and perhaps working in jurisdictions where there would not be regulatory oversight of such experiments. And what was interesting 

was that at that meeting we actually heard about the work that 

was subsequently published for the Chinese group, so it became apparent that, you know, the subject of that conversation was very 

timely. 

I think that, you know, going forward we really have to appreciate the difficulty in putting in place regulations that will be, you 

know, followed internationally. On the other hand, providing oversight and leadership in—by respected scientists in the United 

States and with our international partners I think will be a very 
42 

important thing to do and that’s really what we wanted to achieve 

at that meeting was how to proceed with that sort of approach. 

And I think now having the National Academies involved in organizing larger meetings around this issue is a very desirable outcome. 

Mr. LIPINSKI. Does anyone else want to comment on a temporary 

moratorium on human embryos? 

Dr. DZAU. So the National Academies’ position is that we need 

to be thoughtful, comprehensive, scientifically driven, and independent so we are convening those bodies and obviously supporting 

the idea that we should be very cautious. We have not actually 

taken the position per se because until our work is done, you know, 

we would be looking at what the most objective way to approach 

this when our work is done. 

So I think some of these meetings are critically important and 

important in the sense that we need international scientists to be 

involved with this conversation as well. We have in fact on our advisory board scientists from the Royal Society in London, scientists 

from the Chinese Academy of Science involved with this conversation because it’s really critical to consider all aspects. 

As Dr. McNally pointed out, you know, if you’re using certain 

conditions, I think we have no doubt that this would be enormously 

helpful to patients and to mankind. The question is what are the 

areas we need to be concerned about? 

That being said, I think technology is still early because even 

when you look at the Chinese publication, they were looking at incomplete editing and many other issues that research has to go forward to understand the safety, the off-target effects, and the efficacy before human application. So that’s our position. 

Mr. LIPINSKI. Okay. Dr. McNally? 

Dr. MCNALLY. I would like to echo what Dr. Dzau just said 

which is that I appreciate very much the leadership by Dr. Doudna 

and the group of scientists that got together but I think the IOM 

convening an international effort to really look at what the possibilities are I would think it’s reasonable to wait until we learn 

what that outcome is. It’s important to have a careful and to have 

an international look at what the best recommendations really are 

at this point in time. 

Mr. LIPINSKI. Dr. Khan? 

Dr. KAHN. Just very briefly, the kind of work people are talking 

about on an international level is really critical in establishing 

principles and guidelines for how to move forward. I think moratoria—blanket moratoria are probably not what we need but figuring out when we can go forward and how and under what kind 

of restrictions. So certainly in vitro only and probably, you know, 

for a very long time until it’s determined that the milestones have 

been met in order to move forward into humans. So that’s the good 

work that entities like the Academies can engage in and I think 

critical at the international level. 

Mr. LIPINSKI. Thank you all very much. It’s—yeah, I think there 

are very difficult ethical questions we need to deal with here and 

I know myself I certainly don’t know enough about the technologies, some of the specifics there, and I think that makes—certainly makes a difference. And then we do get to a question that 
43 

Dr. McNally said there are things that we certainly want to cure 

and then the question always is how far do we take this? But we’re 

certainly not going to solve that here so thank you very much. 

Chairwoman COMSTOCK. Thank you. 

Now I recognize Mr. Moolenaar for five minutes. 

Mr. MOOLENAAR. Thank you, Madam Chair. 

And thank you for your presentations today and my compliments 

to you on the work you’re doing. It’s very sophisticated and it 

seems that it has tremendous potential for good. 

On the area of ethics of this I wanted to just explore a few different areas. Dr. Khan, I wanted to start with you. When you’re advising on the ethics of a new genetic technology, are there certain 

ethical lines that should never be crossed, and if so, how are those 

lines drawn? 

Dr. KAHN. That’s a great question and how much time do we 

have I guess is going to be part of my answer. But of course those 

of the first kind of questions that we ask. 

I will say there has been a long-standing line that has not been 

crossed and that is modification of the human germline. That has 

held for decades since the beginning of recombinant DNA technology was available and begun to be implemented. 

One thing that none of us have actually talked about is the recombinant DNA Advisory Committee, which is a committee of the 

advisory to the Director of the NIH, which has in its guidelines a 

statement that they will not consider any proposed research that 

modifies the human germline. So it’s effectively a prohibition from 

that research going forward. Now, it only applies to research that 

is subject to the oversight of that committee, but that has been a 

bright line. 

The other thing which we sort of skirted around but haven’t 

maybe set explicitly is that when there’s such uncertainty about 

the risk and the outcomes, we go slowly. So that’s the kind of a soft 

answer to your question, a mushy one, but I think that’s an important principle to—just to articulate in a way that’s explicit. When 

we know it’s time to go forward is a harder question but we always 

start slowly, especially when we’re talking about potentials for 

modifying humans. 

Mr. MOOLENAAR. Thank you. 

And then, Dr. Doudna, you’re one of the new developers of this 

new technology, and what was it that first sparked your concern 

over the ethics of its use, whether it was in China or when did you 

start being concerned about that? 

Dr. DOUDNA. I guess I realized the potential for this technology 

to operate in the germline first when scientists began to do experiments of that nature in animal models of disease, including mice 

and rats, and then it really came home to me in—it was last year— 

may be almost a year-and-a-half ago that a group again from China 

published a paper in which they had modified the germline of monkeys and made genetically modified monkeys. And that actual monkey model is used very commonly for studying human disease and 

so it seemed very likely at that point that there was no reason to 

think the technology wouldn’t also work in the human germline. 

So, you know, I think we’ve seen now in the scientific community 

that this technology is very democratic in the sense that it works 
44 

across many different types of cells. It doesn’t seem to be limited 

to a particular system. 

Mr. MOOLENAAR. And what role does—you know, I guess one of 

the things that occur to me is the whole area of consent. What role 

does that play in this process when someone is giving consent or 

not? 

Dr. DOUDNA. Are you directing that question to me? 

Mr. MOOLENAAR. Yeah—actually, all four. 

Dr. DOUDNA. You know, I would—maybe I would defer that to 

Dr. McNally. 

Dr. MCNALLY. Well, as a member of an IRB for about 15 years, 

that’s actually—you know, right now, consent in a research study 

in that case would be the parent; the mother would be the person 

providing consent. If a study were to go forward right now, that’s 

who would be providing consent because it would be her materials 

that were being used for that purpose. So there isn’t—from the 

standpoint of human subjects strictly talking like Institutional Review Boards, a fertilized egg is not an individual that provides consent and also even—you know, as a—if it were a child, a parent 

provides consent for that if that answers your question. 

Mr. MOOLENAAR. Yeah. Thank you. 

Dr. KAHN. So it’s—I would sort of ask consent from whom and 

for what? So you’ve heard a version of the answer to that question. 

If we’re talking about the donor of the materials that would be researched upon, that’s one set of questions. If we’re talking about 

modifying an embryo that might one day be implanted into a woman’s body and developed into a child where we have a very difficult 

conceptual problem. How do we think about consent on behalf of 

somebody who’s not yet been born? And so those are the really interesting ethical questions that we will need to confront. We’re certainly nowhere near thinking about doing that kind of application, 

but those are the kinds of questions that need to be identified, articulated, and addressed in efforts that are like the ones the Academies are taking on. 

Mr. MOOLENAAR. I would agree with that. Thank you. 

Chairwoman COMSTOCK. Thank you. 

And now I recognize Mr. Tonko for five minutes. 

Mr. TONKO. Thank you, Madam Chair. 

This hearing shines a light on a difficult but indeed important 

issue and I appreciate my colleagues’ focus on the ethical and legal 

issues surrounding this new technology. 

I’m also assured to see that experts in the medical and scientific 

community are coming together to debate this issue and to discuss 

potential policy implications. However, as we explore the boundaries of what science is capable of and what is ethical and what 

should be legal, we should also take a moment to appreciate just 

how remarkable these advances are. We recognize that these new 

gene editing technologies, including CRISPR, are the outgrowth of 

decades of fundamental research supported by federal agencies, including the National Science Foundation. 

So to Dr. Doudna and Dr. McNally, could you please speak to the 

importance of federal investments in fundamental research, especially the need to support research that may not have any known 

commercial application at the time? 
45 

Dr. MCNALLY. Again, how much time do we have? 

Mr. TONKO. Well, the Reader’s Digest version. 

Dr. MCNALLY. I think everybody sitting here at this table can 

say it cannot be overstated how important the federal investment 

is for research. There are many people who would love to see a lot 

of research funded in the private sector but there are certain aspects of particularly fundamental research that will never be covered in the private sector. Sequencing the genome is a great example of that. 

And we can’t move forward without that federal investment and 

I think all of us here would say, you know, it’s been fairly tough 

times in the last few years with what’s happened with budgets and 

what’s happened with research and watching the effect that that 

has had on the scientific community here in the United States 

where we have actually seen the size and shape of the scientific 

community shrink in the last few years, especially when we look 

across the world and we see it growing elsewhere. So, yes, federally 

funded research is absolutely essential for these types of basic observations. 

Dr. DOUDNA. Right, so I echo everything that my colleague Dr. 

McNally just stated and I want to also add that, you know, there’s 

a tremendous opportunity for the United States to invest in basic 

science. I mean I think traditionally our country is been a leader 

in science partly because we have invested in science that was, you 

know, curiosity-driven research. It was not necessarily targeted on 

curing certain diseases, and I think that we’ve seen again and 

again, especially I would say with regard to technologies, they tend 

to come from unexpected types of projects such as the CRISPR system is a great example of that but there are many others. And I 

think also it’s important to appreciate that commercially, you 

know, these things then have big implications in terms of companies being able to take over and, you know, develop technologies 

that are discovered in academic laboratories but then apply them 

in all sorts of different ways. 

Mr. TONKO. Dr. Dzau, I think you wanted to respond to that, too? 

Dr. DZAU. Well, I totally agree with my colleagues and they’re 

particularly emphasizing support for basic research because if you 

think about this technology, it was done on bacteria, and without 

thinking of application human and look where it is today. And we 

can count so many important breakthroughs that come this way. 

So the ability to support fundamental basic research is critically 

important. 

Also, I think along the issues about saving human lives, creating 

jobs, is our global competitiveness situation, we are truly concerned 

that we don’t continue this level of investment that the United 

States becomes less competitive than many other countries which 

are investing heavily into basic and translational research. 

Mr. TONKO. Thank you. 

Dr. McNally, in your testimony you mentioned how these technologies may be able to be used in somatic or mature cells to treat 

and potentially cure diseases such as sickle cell anemia and muscular dystrophy. Can you please elaborate on this possibility and 

what is that range of therapies and cures that we might only imagine? 
46 

Dr. MCNALLY. I think it’s widely anticipated that sickle cell will 

be one of the first things that’s cured by this where you could take 

a cell out because it’s a bone marrow cell. You could correct it with 

CRISPR/Cas and return that person’s bone marrow cells so it’s not 

a transplant situation; you’re returning their own cells to them. 

And that’s ongoing right now and I anticipate that we will see that. 

For my field I work in muscle diseases. Duchenne muscular dystrophy is a very challenging, challenging area and right now we’re 

looking at technologies where we’re taking small antisense compounds where we would have to treat that individual for a lifetime 

every day with those compounds. Again, if we could get the cells, 

correct them, and reinsert them back in, that would be a one-time 

treatment and a lifetime treatment for that individual. So I think 

we’re seeing a few examples where it’s definitely heading that direction. 

Mr. TONKO. Thank you so much. 

Madam Chair, I yield back. 

Chairwoman COMSTOCK. Thank you. 

I now recognize Mr. Palmer for five minutes. 

Mr. PALMER. Thank you, Madam Chair. 

Several folks have mentioned the research on stem cell. Dr. Tim 

Townes at UAB is a very good friend of mind and doing world-class 

research in that area. 

I’ve got just a few questions. Dr. Dzau and Dr. Kahn, the United 

States and Europe have often disagreed on regulations and policies 

and other areas of biotechnology, for example, genetically modified 

organisms. How does that impact international scientific cooperation? And do you anticipate a similar challenge in human gene editing? 

Dr. DZAU. Well, the intention is that when you get scientists, regulators, ethicists together from different countries, I think responsible individuals would begin to talk about what would be responsible behavior. That I believe is the starting point. And I do agree 

with you that we have some differences in the regulation. But I 

think overall in the issue we’re talking about today, which is the 

application in germline gene editing, I have a sneaking suspicion— 

although I don’t want to fully predict this until the work is done— 

there’s great agreement about the concern about creating successive generations of individuals whose genes have been altered. So 

I have a feeling that we actually will get agreement if not harmonization of many of these thoughts, and certainly it’s our hope that 

we will reach that in our initiative. 

Mr. PALMER. There are current regulations that prohibit federal 

funding for human—for research on human embryos and the FDA 

requires—must issue an investigational new drug application before a biological product may be used in humans. Do you think 

these kind of safeguards are adequate to prevent the kind of experiments that we’re concerned about? 

Dr. DZAU. I’d like Jeff to answer this as well because, as you 

heard, he’s in the midst of leading one of our initiatives on mitochondria DNA replacement. 

Mr. PALMER. Um-hum. 

Dr. DZAU. But I think we very much look into what is the right 

regulatory framework particularly for issues like this, and I would 
47 

say that, you know—and because he would have an opinion but my 

feeling is that this work needs to be done to get better clarity about 

when and how we would regulate some of these areas. 

Mr. PALMER. Dr. Kahn? 

Dr. KAHN. Thank you. In answer to your question, I think that 

the FDA will play a critical role although it will only play its role 

towards the end of the story so the very basic research will be done 

prior to anybody thinking about an IND application or introducing 

it into a therapeutic context. And so we need to think about the 

entire translational pipeline as it were and all of the issues that 

will arise along the way and make sure that we have appropriate 

oversight for each of them. 

The FDA is clearly thinking about the issues that you are and 

are trying to figure out how their framework ought to apply. 

So first—for first in human applications effectively, and then 

once something is licensed, how to control its use and dispersal. So 

one of the things that we all worry about is the so-called off-label 

use of the new technology. So even though the FDA may approve 

it only for a limited purpose, once it’s licensed, it’s hard to control. 

The FDA has some new tools that may actually make them more 

feasible to do and I think as we get closer to the kinds of technology were discussing entering the therapeutic marketplace that 

there will be stronger safeguards in place. 

Mr. PALMER. Well, with these safeguards—and staying on that 

line of thought—is there any worry that if the United States 

doesn’t use this research that we could fall behind our international competition and, you know, could new regulations of this 

technology in the United States put our researchers at a disadvantage or cause them to move their research overseas, Dr. Kahn? 

Dr. KAHN. I’ll let Dr. Dzau and others speak to this, too, but 

there always is talk about that and of course it became a bigger 

issue when the stem cell research—human embryonic stem cell research field really began to grow. And there are now international 

stem cell research societies which try to address issues that are 

clearly not governed by borders. And what we don’t want of course 

is to have people who are doing the best science in the world think 

about leaving this country because it’s easier to do elsewhere. So 

we need appropriate controls but not those that squelch the 

science. Finding that sweet spot of course is the challenge. 

Mr. PALMER. One last question and you can follow up on that as 

you answer this also, but do you believe that we’ll be able to get 

China and the United Kingdom and these other nations to work together to influence change and Europe to adopt similar standards—similar safeguards? 

Dr. KAHN. Maybe Dr. Dzau can speak to that if that’s okay. 

Dr. DZAU. Well, we’re starting with getting the major science 

academies involved so we have the National Academy of Science 

and Medicine in the United States; we have the Chinese Academy 

of Science where you would think that they have tremendous influence on the way the conduct of science is being carried out. We 

have the Royal Society and we intend to include many international bodies. 

So I think the starting point clearly is with the scientists saying 

what would be the right thing to do. One can imagine that we may 
48 

have to escalate this conversation further depending on our findings. 

I just want to point out the question that you asked, which is the 

regulation aspect of this. In fact, you know, we do need a lot more 

clarity in this country. We haven’t talked about the use of gene editing in nonhuman cells, plants, insects, and those changes are— 

that science is really ongoing. We are producing possibly new species that would turn around much faster because of a much shorter 

cycle time, reproductive time. So that regulation also has to come 

in to say what in fact is considered safe and what’s considered as 

environmentally sound. And in fact the National Academies is also 

looking at a study looking at this issue. And as you know, in this 

country the USDA and FDA are involved with animal and plant 

regulations. So we’re also trying to give the right recommendation 

to fortify our regulatory processes. 

The final question you asked earlier about what the right thing 

to do is I think from our perspective at the National Academies I 

think our first issue is human protection and doing the right thing 

for society. I understand of course the potential loss of scientists, 

et cetera, but I do think that we have to actually take the high 

road to say what’s right for us first. And I have a feeling everything else would follow and fall in the right place. 

Mr. PALMER. Thank you. 

Thank you, Madam Chair. 

Chairwoman COMSTOCK. And I now recognize Mr. Swalwell for 

five minutes. 

Mr. SWALWELL. Thank you, Madam Chair, and thank you to our 

panelists. 

My first question relates to something that a number of the 

Members have brought up, which is it seems that America and our 

investments in federally funded research have been in decline, and 

as a result, our successes have been in decline and our ability to 

attract and recruit and retain some of the best and brightest scientists may be in decline. So let’s just for argument’s sake go back 

to 1995. I was 14 years old. And the Human Genome Project was 

just starting to get off the ground and many great results came out 

of that. That was 20 years ago. Would each witness just say more 

or less has America and its—have you seen the investments that 

we’ve made as far as federally funded research, has that made 

America more or less exceptional in this field? So just tell me more 

or less. And I’ll start with Dr. Dzau, just one word. 

Dr. DZAU. More. 

Dr. DOUDNA. More. 

Dr. MCNALLY. More. 

Dr. KAHN. More. 

Mr. SWALWELL. So I’m confused because each of you has said 

that our investments have been on the decline and so you’re telling 

me that we actually have made more investments since 1995 and 

you believe we are more exceptional now in these fields? 

Dr. MCNALLY. You picked 1995. 

Mr. SWALWELL. But comparing—— 

Dr. MCNALLY. If you said 1995—— 

Mr. SWALWELL. Sure. 
49 

Dr. MCNALLY. —to 2005, we would say more. If you would say 

2005 to 2015 we would all say less. 

Mr. SWALWELL. Is that right, less, Dr. Dzau? 

Dr. DZAU. [Nonverbal response. Nodded in the affirmative.] 

Mr. SWALWELL. Dr. Doudna? 

Dr. DOUDNA. [Nonverbal response. Nodded in the affirmative.] 

Mr. SWALWELL. Dr. McNally? 

Dr. MCNALLY. [Nonverbal response. Nodded in the affirmative.] 

Mr. SWALWELL. Dr. Kahn? 

Dr. KAHN. [Nonverbal response. Nodded in the affirmative.] 

Mr. SWALWELL. Okay. So that was my question. You would agree 

that we have become less exceptional in the field of genetic engineering as far as it relates to human DNA since 1995, that we’ve 

been on the decline? 

Dr. MCNALLY. 2005. 

Mr. SWALWELL. 2005 is the—— 

Dr. MCNALLY. Yes. 

Mr. SWALWELL. —point, Dr. McNally? 

Dr. MCNALLY. Yeah. 

Mr. SWALWELL. So what’s exciting about this research and this 

field is the potential for us to conquer diseases before they conquer 

us. And I look at the example of Huntington’s disease, which affects anywhere from 30,000 to 200,000 people, and it’s a disease 

that is so cruel it steals your memory and affects your muscular 

system. 

And I’m just wondering, maybe if Dr. Doudna can tell us and 

have others weigh in, what can the United States do specifically to 

take leadership in this area if we have the appropriate funding so 

that we can conquer these diseases? 

Dr. DOUDNA. So I think I know, what—again sort of maybe echoing something that I mentioned earlier and that has been discussed 

here, I think, you know, the United States has been a real leader 

in basic research for a while and all of us are concerned that we 

see that edge slipping away over time. And so I think that, you 

know, the investment in fundamental research that will allow scientists to understand, for example, genome engineering technology 

like we’re discussing today, how does it operate, how can we deliver 

it to patients, how do we ensure that it’s operating as we intend 

and not creating unintended consequences, that it’s safe, that it’s 

effective. 

All of those lines of research are going to require, I would say, 

a combination of efforts by people like me that do basic research 

and people like my colleagues who are medical doctors and think 

about clinical issues. We need to be putting our efforts together 

and that has to be I think supported by federal funds. 

Mr. SWALWELL. And how can we tell the story to the American 

people who look to us as a Congress to make the decisions when 

it comes to funding with so many competing priorities? How can we 

tell them that something—like $1 invested in basic research where 

you may not be able to tell us what disease you’re going to be able 

to cure 10 to 15 years from now but there’s still—the taxpayer is 

looking to us to, you know, hold accountable the funding. Like how 

can we better tell the stories of the science community about what 

we could see from this down the road and how we could truly, you 
50 

know, attack and cure some of these diseases? I think that’s probably one of the biggest challenges. And maybe Dr. McNally would 

want to answer. 

Dr. MCNALLY. Yeah, I mean we’ve seen incredible advances that 

have come out of basic research, and I’ll talk about my field, which 

is cardiology. It was out of basic research of understanding the 

LDL receptor that led to statins, the drugs that probably a lot of 

people in this room take, and we’ve seen a direct translation to a 

reduction in the rate of heart attacks. I mean it’s a very different 

world than what it was when I first heard of my training 20 years 

ago. We don’t see acute heart attacks like we used to and that 

came as an outcome of basic research not that many years ago. 

So we can tell the same story for heart disease. We can tell the 

same story for many cancers. Cancer and heart disease are the 

major things that kill people so we’ve made huge headway in that. 

Mr. SWALWELL. Great, thank you. And, you know, I know every 

Member up here has thousands of people in their district who go 

to bed on their knees praying that people like you will make discoveries that will make them or their relatives live healthier lives. So 

thank you for what you’re doing and hopefully we can do the right 

thing here and better fund your initiatives. 

And I yield back. 

Chairwoman COMSTOCK. Thank you. 

And I now recognize Mr. Westerman for five minutes. 

Mr. WESTERMAN. Thank you, Madam Chair, and thank you, 

panel, for your invigorating testimony. 

Dr. Dzau, what’s your anticipated timeline for the initiative on 

human gene editing? 

Dr. DZAU. We are convening an international summit in the 

fall—late fall and that should be a—I think really an important 

meeting that will get together international scientists. As you 

heard from our previous experience at Asilomar in the 1970s, you 

know, one would engage in the discussions that Dr. Doudna and 

McNally and Kahn put forth, and hopefully the findings of that 

meeting will be published very shortly after that. 

Perhaps equally important is a concurrent deep-dive study which 

we’re conducting that would involve analysis, research, the assessment of risk/benefits, as well as a regulatory framework and ethical 

issues, and that usually—what we call a consensus report would 

take as long as about a year, although we’re hoping that we would 

try to move it faster for exactly the reasons that we’ve been talking 

about. Such a report with very specific recommendations which 

we’re willing to put forward under both public hearing and also 

closed discussions will be available to you, to the Nation, and to 

many others. So I could say the time frame is late fall to sometime 

next year but hopefully as early as we possibly can to put out that 

report. 

Mr. WESTERMAN. So how great are your concerns that the initiatives may not be able to keep up with the breakneck speed of the 

technology as it moves forward? 

Dr. DZAU. This is exactly why we need a summit, a meeting first, 

where key scientists—and we’re going to engage a large number— 

will discuss about what would be considered as good conduct, good 

oversight, understanding risk, et cetera, so that the scientific com-
51 

munity which is really driving most of this research understands 

those issues and have in general agreement. 

We also believe of course in our study itself that becomes a definitive document by which you and others can be informed to say 

what are the right decisions based on consensus. 

Mr. WESTERMAN. So you said the scientific community is driving 

the research. As the National Academies’ work on the new initiatives for building the framework, do you believe the scientific community will embrace and voluntarily follow the guidelines or will 

there need to be regulations or laws put in place? 

Dr. DZAU. Well, you know, as you already heard, I think there 

are many responsible scientists, particularly the ones who are leading this field, who feel already that we should, you know, put a 

moratorium and not slow it down. So I do believe that already 

going into this meeting, although there may be many different 

opinions, there’s probably general agreement that we need to slow 

down this area until we have a much clearer point of view about 

where we should be going and the clarity in terms of regulation. 

The problem I’m concerned about is that we rush into this too 

fast. We don’t really want to and it’s such an important issue. 

We’ve got to be very thoughtful. That being said, we understand 

the time urgency of the issue—situation. 

Mr. WESTERMAN. All right. And the advisory group to the initiative that was named yesterday, it includes scientists and researchers from China and the United Kingdom. Do you believe these participants will help influence Asia and Europe to adopt similar 

standards? 

Dr. DZAU. We certainly hope so, and in fact I was on the advisory 

group. Our intention is that in an international summit we include 

a lot more scientists from Asia and every part of the world, Europe, 

et cetera, to be part of this discussion. 

You know, it’s interesting when we think back on U.S.’s position. 

You know, when the Asilomar Conference come about, the United 

States was the main show in town about technology, so among the 

U.S. scientists, you can imagine there’s agreements, you know, so 

there’s a general way of saying, you know, what do we do next? 

Here, we really need to include international scientists. 

Mr. WESTERMAN. So what capacity and infrastructure does the 

Chinese Government have in place for regulating human scientific 

research? 

Dr. DZAU. You know, I’m not that familiar with the regulation 

at this point in China. As you heard from my colleague, Dr. Kahn, 

there is some speculation over what’s there and what’s not there. 

I think we would hope that that meeting will bring out with clarity 

what each country’s position is, what’s the regulatory position, 

what’s the scientific position so that we can all come together and 

examine this together. 

Mr. WESTERMAN. Thank you, and I believe I’m out of time, 

Madam Chair. 

Chairwoman COMSTOCK. All right. Thank you. 

And I now recognize Mr. Foster. 

Mr. FOSTER. Thank you, Madam Chair. 

In addition to the international summit that you’re having this 

fall it strikes me that a full-blown National Academies study may 
52 

have merit to do a deeper dive into this. And so, Dr. Dzau, would 

a letter signed by Members of Congress, for example, help you in 

recruiting assistance for this sort of effort? 

Dr. DZAU. I think it would be outstanding in fact if we got a letter from Congress on this. We took this upon ourselves as a National Academy because that’s what we do, and we know it’s the 

right thing to do. We have the support of scientists and others to 

say go forward. But I think it would be tremendously impactful if 

Congress would provide us with that kind of support a mandate to 

go forward with this. 

Mr. FOSTER. Okay. Just a quick question, today what’s the rough 

cost and time to get a mouse model with a specific genetic modification? Does anyone—just roughly within a factor of two—you know, 

is it $10,000 or $5,000 or—— 

Dr. MCNALLY. Yeah. Well, with CRISPR/Cas initiated, yeah, 

you’ll probably decrease the cost in half and decrease the time in 

half. So if it was $25,000, it’s probably closer to 10 now, and if it 

was a year, it’s probably closer to 6 months. That’s counting breeding time. 

Mr. FOSTER. Well, when—so one of the things slowing down the 

application of this is a lot of—sort of two classes of worries about 

potential dangers. The first is so-called off-target effects where, in 

addition to the genetic modification you want, you get inadvertent 

modifications to the genome. And it’s my understanding that the 

technology is evolving rapidly and that—I was just wondering if 

you—I’ll ask you to go out on a limb I guess, and Dr. Doudna first, 

about—you know, if you just look at the rate of progress on this, 

what is the rough timescale where we can expect—where we might 

expect you’ll be in a position that it could be used, you know, ‘‘safely’’ on humans? 

Dr. DOUDNA. Well, I think one has to, you know, sort of distinguish what types of applications we’re talking about. I think if 

we’re considering application to treat a disease like sickle cell anemia where the editing could be done on cells that are taken out of 

the patient and then validated before they are reintroduced into 

the body, I feel that that is likely to happen, you know, the next 

year or two honestly. I think it will be very—— 

Mr. FOSTER. Right, so no technological development there? 

Dr. DOUDNA. No, because I think we already have the ability to, 

you know, validate the correct sequencing—correct editing was 

done by DNA sequencing in that sort of a scenario. I think if you’re 

talking about an application like, you know, we want to introduce 

the tool into a patient’s body and where you want editing to occur 

in the body, then we’re—that’s further off. First of all, we don’t 

have the—very good ways to introduce this into specific tissue 

types yet, and also we don’t have good ways to validate that the 

correct type of editing was done without off-target effects, as you 

implied. But I think for any kinds of applications where we can do 

the validation outside of the body, that’s going to move forward in 

the next year or two. 

Mr. FOSTER. Okay. And then if I raise the stakes further to 

germline editing, is that something that may just never happen? It 

may never be reliable enough? Or is it a reasonable guess that 

within the next five years that you’ll be able to validate the 
53 

germline editing, that it has taken place correctly and with high 

enough confidence that— 

Dr. DOUDNA. Well, I’m very interested to hear my colleagues’ answer to that question but I guess my answer would be that it will 

depend on the way that research is enabled around, you know, that 

sort of application. I think if it’s possible to do experiments in germ 

cells so that we can understand how this technology works, operates in those types of cells, then I think, you know—boy, it’s always 

hard to put a timeline on things but, you know, certainly within 

a few years it’ll probably be to the point where one could, you 

know, employ it for that sort of application. But I don’t know if 

you—my colleagues would agree with that or not. 

Dr. MCNALLY. I agree. I think that’s a reasonable timeline, five, 

ten years if you had to guess. 

Mr. FOSTER. Yeah. I’m trying to, you know, get some idea—— 

Dr. MCNALLY. Yeah. 

Mr. FOSTER. —of what the response time from Congress and our 

society has to be for that. 

There’s a second class of potential dangers having to do with just 

misunderstandings about what the effects of a specific genetic 

change will be on the characteristics of the adult organism. And, 

you know, over the spread of, you know, different things from simple conditions like sickle cell to complex things like, you know, personality, you know, what is your guess for the timescale that we’re 

looking at there from right now to never? 

Dr. DOUDNA. My answer is certainly much longer. I think we— 

I think—and that—to me that’s not limited by the genome editing 

technology as much as it’s limited by our knowledge of the human 

genome. 

Mr. FOSTER. Okay. Thank you. My timer has gone down. I yield 

back. 

Mr. MOOLENAAR. [Presiding] Thank you. I now recognize Dr. 

Abraham. 

Mr. ABRAHAM. Well, after I read you all’s testimony last night I 

went on and read each of you all’s bios. Your parents must be very 

proud. 

We all in this room I think understand the potential that this 

type of research can lead to not only in the human endeavors but 

in plant technology, curing world hunger. So it’s applicable to so 

many aspects of humanity. 

And to—I think it was Dr.—your point, Dr. McNally, that it 

probably would be unethical if we have a child with sickle cell in 

the ALL and this therapy is available not to offer it. 

Dr. Doudna, I salute your intelligence for recognizing it even 

though I know you weren’t particularly looking for the CRISPR/ 

Cas9 technology that you recognized it. I compared it last night— 

I was reading—to Fleming discovering penicillin. He had been—I 

think maybe this CRISPR can save as millions of lives as penicillin 

has, so kudos to you guys for what you do. 

I guess the question I’m leading up to, the old adage if you get 

two doctors in a room, you get three different opinions, as we know 

very true. That’s certainly on my end of the stick. 

Dr. Doudna, you published I think an article in a science magazine that you wanted this moratorium and, Dr. Dzau, you’ve been 
54 

pushing for. Are you guys getting some pushback from members of 

your community that says, you know, no, we don’t need a moratorium and let this thing go? Let this genie out of the bottle and 

don’t put it back in? 

Dr. DOUDNA. Well, I can tell you what I’m seeing. I think that, 

you know, at around the same time that we published the perspective in Science magazine a related perspective was published in 

Nature magazine from a different group that actually called for I 

would say real moratorium even on research. So that group basically was advocating not proceeding with any kind of research on 

human germ cells using genome editing technology. And I just 

want to point out that in the group that I met with in January, 

we actually discussed that and felt that actually we—in our opinion 

research on those types of cells, appropriately regulated, should be 

enabled, just not clinical application. 

Mr. ABRAHAM. Dr. Dzau, when—Dr. Kahn, when you had the— 

I think it was back in 1975 I was reading last night y’all had a recombinant DNA moratorium that you tried to put forth voluntarily. 

In the international community, was that followed? Has that been 

pretty much adhered to throughout the last few generations? 

Dr. KAHN. So a little bit before my time but it’s a landmark in 

the area of science policy and then it was a voluntary moratorium 

instituted by the scientific community. The truth is we haven’t seen 

anything like that since. It was a very important undertaking. And 

I think when the scientific community got together to talk about 

the implications of recombinant DNA technology at that time, they 

weren’t sure whether the scientific community would follow what 

the—— 

Mr. ABRAHAM. Much like now. 

Dr. KAHN. Much like now. It’s 40 years later and the scientific 

community is very different today than it was in 1975. 

Mr. ABRAHAM. For sure. 

Dr. KAHN. So we were undisputedly the leaders in the world of 

that science in 1975. As everybody has said, the technology now is 

much more widespread and much easier to implement, as Dr. 

Doudna has said, making it much more difficult for any one community of scientists to actually speak on behalf of the whole. 

Mr. ABRAHAM. Well, we think—we can probably say realistically 

that America will lead in the discussion of the ethical and moral 

implications of this, but to follow up with your statement then, as 

Americans or as the United States—— 

Dr. KAHN. Um-hum. 

Mr. ABRAHAM. —scientific community, if we should see an element get outside the bounds, can we do anything? 

Dr. KAHN. Yeah, it’s a great question. And as I said in my testimony and in my statement that you read and I reiterated today 

that the journal publication community has a very important role 

to play here. What—your research doesn’t really mean much unless 

your peers have reviewed it, called it good, and then it gets published in a credible place. So that’s a really important barrier. That 

doesn’t mean people won’t try to do things that aren’t ethical and 

then try to get them published, but it’s a—has a sort of strong 

inoculating feature I would say. 
55 

The other thing that Dr. Doudna pointed out but I’ll reiterate is 

that there’s been a little bit of a disagreement in the scientific community on the topic of gene editing, about whether there should be 

a moratorium only on clinical application or on something more 

widespread. And so that’s a healthy debate to have and it’s great 

that we’re having it and it’s great that the Academies are bringing 

it out to the international level. So that’s exactly the discussion we 

ought to be having. 

One last thing I’ll say is that people around the world want to 

be part of the scientific community. There’s a very strong incentive 

for them to behave, right, to follow the conventions which make 

them a legitimate member and I think we shouldn’t underestimate 

the power of that. So, yeah, there might be fewer restrictions in 

other parts of the world but those people want to publish in Science 

and Nature just like American scientists do. 

Mr. ABRAHAM. Yeah. Thank you. 

Mr. Chairman, I’ll yield back. 

Mr. MOOLENAAR. Thank you. 

I now recognize Mr. Sherman. 

Mr. SHERMAN. Thank you, Mr. Chairman, for holding these hearings. This has been an area of intense interest on my part since 

the year 2000 when I went to the Floor and said that the most important decision we will make this century is whether our successor species is carbon-based or silicon-based, whether the new 

and intelligent species on this planet is the product of genetic engineering or the product of computer engineering. 

Some of you will remember I served on this Committee in the 

107th and 108th Congresses and this was pretty much my main 

reason for serving on the Science Committee. 

I should bring to the attention of this Subcommittee that on June 

19, 2008—transcript available—the relevant Subcommittee of Foreign Affairs had a hearing titled ‘‘Genetic and Other Human Modification Technologies: Sensible International Regulation or a New 

Kind of Arms Race?’’ And in fact the analogy to what we’re talking 

about here is the only other technology that was equally explosive 

perhaps, and that is nuclear weapons technology. In 1939 Albert 

Einstein wrote to Roosevelt saying what was possible and policymakers had only six years before that technology literally exploded 

onto the scene. Thank God we’ve got a little bit more time but the 

Nonproliferation Treaty took many decades after 1939 or after 

1945 and I think could be a good model for what we need here. 

Dr. Kahn, it may be too long to list but I don’t know whether 

America is exactly number one in this technology or whether Britain or China might be slightly ahead, but we’re all within, I think, 

a few years, but there are a whole bunch of other countries either 

at that level or maybe four or five years behind. Can you even list 

the countries that within five years could be where we are now? 

Dr. KAHN. I don’t know— 

Mr. SHERMAN. Are we talking a dozen, two dozen? 

Dr. KAHN. I’m not sure that it’s—that I or anybody could do that, 

and in fact, I’m not sure that it even requires countries. It’s individuals who have access to the capacity— 

Mr. SHERMAN. Um-hum. 
56 

Dr. KAHN. —which, as Dr. Doudna has said, is actually fairly 

democratic I think was the term that she used. So in a way, you 

know, it’s about where people have the laboratory capacity and 

that’s almost anywhere in the world. 

Mr. SHERMAN. Well, thank God nuclear weapons take an industrial scale. And although they reflect only 1945 technology, we’ve 

seen in Iran that you have to do something big, you have to spend 

billions of dollars, it has to be visible to the world that you’re doing 

something. You seem to be saying that what we’re concerned about 

here could be a lot cheaper and take place in a laboratory basement? 

Dr. KAHN. And I’ll let my colleagues speak to the concrete answer to that question but I think exactly that kind of point is really 

important for how we think about what appropriate oversight, regulation, guidelines need to be— 

Mr. SHERMAN. And I want to pick up on Mr. Foster’s question 

about time frame, but one of the concerns we will have is that 

countries will see this as, oh my God, it might be terribly unethical 

but it gives us a leg up militarily or economically. Damn the torpedoes. 

Leaving aside engineering intelligence and looking to things that 

would—other than that that would affect soldiers, soldier is a little 

better if they’ve got courage, stamina, and strength. We’re already 

at a point where drugs are going to be used by various militaries 

to impart those characteristics to their soldiers but then we can go 

further to genetic engineering. What is the time frame before 

there’s genetic engineering that would do the simplest of—I don’t 

know which of those three is easiest—would give a soldier either 

more strength or more stamina or, say, more courage, more willingness to charge out? Is there any way to say that that soldier is five 

years from now, fifteen years from now, or are we in the world of 

science fiction? Anybody venture a guess? 

Dr. MCNALLY. Science fiction. 

Mr. SHERMAN. Okay. Well, we’ll be in a position I think next decade where at least—well, already many militaries are using drugs 

on their soldiers and then the next step will be to use the next element of medical technology, not drugs, but genetic engineering. 

Does anyone have—I mean we’re—the first and most ethical use 

of this technology is to remedy maladies. The next step will be to 

allow parents to have kids that have all the best characteristics of 

anyone in their family or in the world. And then we’ll go to giving 

kids unprecedented capacities, and at that point we’re talking 

maybe human, maybe trans-human. What—does anyone here have 

any view as to how long it will be before we can affect the intelligence of either people—either adults or of germ cell—the germ 

line? 

Dr. MCNALLY. I’ll dive in. 

Mr. SHERMAN. Dr. McNally is coming close. 

Dr. MCNALLY. I’ll dive in. 

As Dr. Doudna has said, the limitations of engineering things 

like intelligence— 

Mr. SHERMAN. Um-hum. 

Dr. MCNALLY. —are far more limited by our genetic observations— 
57 

Mr. SHERMAN. Um-hum. 

Dr. MCNALLY. —than they are by our capacity to do genome engineering. We have actually very large scale genetic projects ongoing right now, the 1,000 Genomes Project in the next year or two 

we’ll be looking at a million human sequences and a lot of information connected to it. And the simple answer is that traits like intelligence are not single-gene— 

Mr. SHERMAN. Yeah. 

Dr. MCNALLY. They’re probably not even entirely genetic is what 

people are referring to on a regular basis right now. There’s a 

whole series of articles written about the missing heritability for 

many different traits, things that we thought were genetic when it 

turns out we look at the genes, they may not be so genetic or they 

are a very high complexity. 

Mr. SHERMAN. So the technology—it’s interesting because in nuclear weapons there’s two components. One is weaponizing the 

highly enriched uranium, which turns out to be the easy part. It 

seems the most dangerous. Oh, my God, you’re going to turn it into 

a nuclear weapon. The real hard part and the decider as to which 

countries have nuclear weapons technology is the ability to enrich 

uranium. 

Mr. MOOLENAAR. The gentleman’s time is— 

Mr. SHERMAN. If I—it seems like what you’re describing is a situation where the roadmap to what genes do—have what characteristics is the hard part and the part that will determine and the actual snipping and replacing and editing, you guys have that down. 

I yield back. 

Mr. MOOLENAAR. Thank you. 

Ms. Bonamici. 

Ms. BONAMICI. Thank you very much, Mr. Chairman. And thank 

you to the Chairman and Ranking Member for holding this hearing 

about this important topic, and I—my absence for most of the hearing was only because I was in another hearing. It does not indicate 

my lack of interest in the subject. 

And I’m really glad that we have this, I agree, very incredibly 

qualified panel and I especially appreciate the gender diversity. As 

someone who works on education issues and trying to get more 

women in STEM, thank you for having a balanced panel. 

So I missed—a lot of the questions have already been asked but 

there’s a couple of things that I wanted to follow up on. There’s 

been a lot of attention on using gene editing technologies in human 

embryos, and of course we—with the press from what happened in 

China, that’s getting attention. But I know that a lot of the research is not in human embryos, so could you discuss how the technologies are being used in research today in other areas, including 

in organisms other than humans? And also can you talk about the 

potential promise from sectors other than healthcare, energy, for 

example? 

Dr. DOUDNA. Okay. Well, I’ll—I can take a stab at that. So you’re 

absolutely right that the technology is being widely employed in 

many different kinds of cells and organisms, and I’ll just give you 

a couple of examples. I think that in plant biology this is going to 

be, you know, equally impactful as the kind of thing that—kind of 

applications that we’re talking about here in human health in 
58 

terms of enabling very, you know, widespread introduction of genes 

into plants that could be beneficial especially for dealing with climate change and other kinds of environmental impacts in plants. 

And I’m just—I’m not a plant biologist but I’m saying this based 

on conversations I’ve had with people that are already doing those 

kinds of experiments and are extremely excited about the way that 

that research has been enabled. 

And then you mentioned biofuels and I think that’s very interesting because I’m aware of several groups that are actually using 

this kind of genome engineering for what we call systems biology, 

basically being able to make large-scale changes in the genomes of 

organisms that will be useful for producing various kinds of chemicals, including, you know, biofuels and other very important materials that can be difficult to obtain in other ways. 

Ms. BONAMICI. Terrific. And I know Dr. Dzau wants to—— 

Dr. DZAU. This in fact is a very important area. Today we’re talking about human genome editing but I’m glad you raised the question about the usage in other living organisms. Mr. Sherman asked 

about, you know, what will be the misuse, if you will, but we do 

have to think about, you know, the use in plants, insects, et cetera, 

you know, what happens if there’s misuse and what would happen 

to the environmental impact and who actually gets to decide who’s 

going to do what? And there are more commercial sources I believe—opportunity for commercialization in changing coffee beans 

or whatever that you can imagine. 

So this is an area that in fact—called gene drive that, again, National Academies are looking very carefully at this and how to regulate this. So I do think this is to me a very important area even 

though our focus is on human gene editing. 

Ms. BONAMICI. Terrific. And I’m going to slightly change the 

topic. 

Dr. Doudna—did I say your name properly? 

Dr. DOUDNA. Yes. 

Ms. BONAMICI. I know you cofounded several startup companies 

and we’ve had conversations in this Committee before about the 

challenges in trying to launch companies but also transferring academic research into the marketplace. So can you chat a little bit 

about that and what the challenges have been and what the Federal Government can do to help with transferring academic research into the marketplace? 

Dr. DOUDNA. Yeah, this is a very important area and I’m a real 

newbie to it. This is actually the first time that research in my lab 

has led to something that, you know, had clear commercial applications. So I can tell you my experience, you know, being involved in 

starting companies and raising money for companies. We’ve had 

different experiences I would say. I’ve been involved in three different startups around this technology so far. And I’ve had a lot of 

help with one of them in particular through a—what would I call 

it? I guess it’s a biomedical research initiative in the Bay area that 

was actually funded in part by the State of California. And what 

that does is to give people like me who know nothing about, you 

know, starting a business some training. We got access to some 

legal advice early on. We—I think this Institute paid for the incor-
59 

poration of the company initially and then gave us some support 

in terms of writing for federal funding for the company. 

I think this kind of support is really important. And as I talk to 

my colleagues around the country who are trying to do similar 

things, commercializing work coming out of their academic labs, I 

hear over and over how all of us who are in the academic world 

could really benefit from that kind of level of support. I’m not sure 

if it’s something that happens at the federal level or if it’s better 

done lower down, hard to say. 

Ms. BONAMICI. Thank you so much. 

And my time is expired. I yield back, Mr. Chairman. Thank you. 

Mr. MOOLENAAR. Thank you. 

And now I want to recognize Mr. Palmer. 

Mr. PALMER. A couple of quick questions. Most of this discussion 

I think has been about government research. You’re talking about 

convening an international summit. I just want to make sure that 

when we’re talking about implementing safeguards that this includes the private sector, that they’re not running out there as 

mavericks. Is that correct? 

Dr. DZAU. Certainly our intention, if you look carefully at the 

way we put our advisory committee together, we have people who 

have great industry experience on it and our intention is to include 

industry and, you know, people in the commercial sector to be in 

this discussion. 

Mr. PALMER. Thank you. I yield the balance of my time, Mr. 

Chairman. 

Mr. MOOLENAAR. Thank you. 

I now recognize Mr. Foster. 

Mr. FOSTER. Thank you. 

It’s my understanding that very often there’s a computing bottleneck in our ability to analyze genomes. And I guess, Dr. McNally, 

can you tell the Committee about some of the work you’ve done at 

Argonne—— 

Dr. MCNALLY. Yeah. 

Mr. FOSTER. —which is a facility shared by the Ranking Member 

and myself. 

Dr. MCNALLY. Yeah, so right now it’s—since more than a yearand-a-half, it’s been very possible to sequence a human genome 

with the consumable cost being about $1,000, which is an amazingly low price for a single genome. That doesn’t include the cost 

of analysis, and so the real rate-limiting step right now is the time 

it takes to analyze genomes. 

So what we’ve done is we’ve actually, working together with Argonne National Labs, the University of Chicago, and now Northwestern, we’ve actually taken a Cray XE6 supercomputer that’s at 

Argonne and outfitted it with all the computing code that’s available through the Broad Institute so that we can now, for example, 

analyze 250 genomes in a weekend if they give us the whole machine to work. So that dramatically accelerates our ability to screen 

through three billion bases of an individual genome and score it’s 

four million differences that exist in each one of those genomes, the 

vast majority of which are rare and private. 
60 

Mr. FOSTER. And—well, thank you. That’s very interesting. I 

guess another application of federal investments that are having an 

unintended benefit. 

I guess for Dr. Kahn, has anyone ever taken the bull by the 

horns and actually attempted to draft legislation or international 

treaties, you know, at regulating human genetic engineering or 

other genetic engineering in the environment? 

Dr. KAHN. That’s a good question. I don’t know that there have 

been legislation or, you know, bills proposed and I don’t know 

about international treaties. There certainly have been efforts to 

craft guidelines but they’re nonbinding by entities like the World 

Health Organization or the World Medical Association, which has 

crafted actually fairly well-known guidelines on human subjects research which is called Declaration of Helsinki, which tends to be 

followed but in a voluntary way and then built into regulation at 

the national level. But I don’t think that there has been any successful international regulation. 

Mr. FOSTER. All right, or even proposed—just—you know, lists 

enumeration of all of the issues that you have to resolve when you 

write rules. 

Dr. KAHN. Yeah, I’m not aware, although not a historian of 

science. That’s a really interesting question that I—maybe I’ll do 

some looking and see if I can get back to you. 

Mr. FOSTER. I would appreciate that. 

Dr. KAHN. Sure. 

Mr. FOSTER. And do you understand right now if you’re on a hospital ship in international waters, who’s the regulator? 

Dr. KAHN. That’s the right kind of question to be asking. And 

this sort of issue came up when there was a supposed cloning of 

a human being. You may remember back in—when that—whenever 

that was, the late ’90s, early 2000s— 

Mr. FOSTER. All the—— 

Dr. KAHN. Yeah, which turned out to be a hoax. But one of the 

claims was it was being done in international waters and out of the 

reach of any of the national regulations or governance structure, 

which, you know, in a way was a helpful aha moment, right? You 

need to think about what to do when all you need is a well-fitted 

ship that has the laboratory on board and you’re outside of the— 

any restrictions that an international treaty or national government might employ. 

Mr. FOSTER. Okay. And I guess one last question. The issue of 

gene drives has entered the news and interestingly is with the potential to sort of take over the genome of an entire species in the 

wild in the course of, you know, a few dozens of generations. And 

so this obviously has huge environmental effects and has to be 

internationally regulated presumably because of, you know, insects 

don’t normally, you know, obey national borders. 

And so I was just wondering if that is an area where, for example, this Committee might interestingly have a separate branch of 

investigation that all of the applications of this technology to plants 

and animals in the wild and in the laboratory? 

Yes, Dr. Dzau. 

Dr. DZAU. Mr. Foster, as I mentioned, I think the National Academies is undertaking such a study. We’d be happy to send you all 
61 

the materials, and in fact, as we look at what we are potentially 

covering and not covering, we can have a conversation about what 

else should be done. 

Mr. FOSTER. Okay. And so the charge is complete for the study 

or is it—— 

Dr. DZAU. Yes. 

Mr. FOSTER. —ongoing? All right. I’d appreciate that. Thank you. 

I yield back. 

Mr. MOOLENAAR. Thank you. 

And I’d like to thank our witnesses for the testimony today, outstanding, and all the Members for the questions. The record will 

remain open for two weeks for additional comments and written 

questions from Members. The witnesses are excused and this hearing is adjourned. 

[Whereupon, at 3:56 p.m., the Subcommittee was adjourned.] 

(63) 

Appendix I 

ANSWERS TO POST-HEARING QUESTIONS
64 

ANSWERS TO POST-HEARING QUESTIONS

Responses by Dr. Elizabeth McNally 
(65) 

Appendix II 

ADDITIONAL MATERIAL FOR THE RECORD
66 

STATEMENT SUBMITTED BY FULL COMMITTEE RANKING MEMBER

EDDIE BERNICE JOHNSON

Thank you, Madam Chairwoman for holding this hearing, and I 

want to join you in welcoming our distinguished panel of witnesses. 

This afternoon we are talking about new gene editing technologies that have promising applications in fields ranging from 

medicine to energy to agriculture. The Chinese research paper that 

prompted this hearing highlights the need to have a serious examination of the science, safety, and ethics of gene editing technologies. 

I want to thank the Chairwoman and Ranking Member for putting together this hearing with an impressive panel of expert witnesses. Additionally, I want to acknowledge Dr. Foster, whom I understand was instrumental in advocating for this hearing. 

The technologies we are discussing today can alter DNA—the 

blueprint of life. There are significant safety, efficacy, and ethical 

issues concerning these technologies. What are the ethical uses of 

these technologies? Is it ethical to use them if they could cure a disease? What if they just treated a disease? 

Any applications that would alter germline cells, where changes 

are passed down through generations, have additional ethical 

issues. For example, is it ethical for the current generation to consent to changes for a future one? 

Although today we are discussing human applications, we should 

not forget that these same technologies also have great potential 

for use in energy and agriculture. Gene editing could be used to 

create biofuels and new crops. Responsible applications of these 

technologies could lead to significant economic growth if the U.S. 

takes the lead in research and transferring that research to the 

private sector. 

I look forward to hearing from the witnesses about the state of 

the gene editing science and potential applications. Additionally, 

the U.S. needs to take a leadership role in addressing relevant ethical issues so I am glad that the panel includes a bioethicist to help 

us better understand what is involved. 

Finally, I look forward to hearing about the National Academies’ 

plans to address these and other important questions surrounding 

this emerging research area. 

Thank you and I yield the balance of my time. 

Æ