A Gene Therapy Institute for NIH?
Authors: Inder M. Verma
Journal: Molecular Therapy (2002)

On December 20, 2001, the 107th Congress gave final approval to $23.6 billion of funding in fiscal year (FY) 2002 for the National Institutes of Health (NIH). This healthy increase of $3.2 billion (15.7%) over FY 2001 is on track to fulfill the bipartisan goal of doubling the NIH budget between 1998 and 2003. Every institute at NIH received an increase greater than 12%, and several did even better. The NIH is a confederation of many institutes, each with its own director, administration, and mandate to promote mission-oriented research. Most of the individual institutes at the NIH support and fund some form of gene therapy research. It is difficult to measure the exact amount, but I estimate that the combined efforts of the NIH in the area of gene therapy amount to $300–400 million per year. Given this, why not establish a National Institute of Gene Therapy Research (NIGTR)? When the idea of sequencing the entire human genome was first touted in earnest in the early 1980s, the National Human Genome Research Institute (NHGRI) was established. We all know its seminal role in establishing genome centers, mapping the genomes of not only human but of many species, and eventually deciphering the entire nucleotide sequence of several species. NHGRI provided the impetus to develop new technologies, promoted large-scale automation, brought together diverse experts, and trained a new generation of scientists who can combine traditional genetics with bioinformatics to mine the immense data buried in these billions of A's, T's, G's, and C's. The annual budget of NHGRI for FY 2002 is $430 million. The NIGTR could focus on providing resources for generating new delivery vehicles that are efficient and safe rather than responding to requests for applications often put forward by individual institutes for the disease in which they are most interested. NIGTR would support a program dedicated to identifying tissue-specific promoters, enhancers, insulators, motifs necessary for message stability, and elements needed for efficient translation. The NIGTR could offer resources to bring together virologists, structural biologists, and biophysicists to generate targeted delivery vehicles. It will encourage gene therapists to collaborate with immunologists to understand and manipulate the world of the immune system. I can imagine a growing role for NIGTR in providing resources for the field of stem cell biology: If we can transduce stem cells that can then be transplanted and undergo in vivo expansion to make functional tissue or organ, a whole new application of gene therapy will emerge. What about tissue engineering and therapeutic cloning? NIGTR can make a concerted effort to offer inducements to scientists to develop novel systems to regulate transcription and eventual production of the transgenic product. Gene therapy will have an impact on nearly all aspects of human health. Why inject erythropoietin (EPO) daily or weekly when a gene producing regulated amounts of EPO can be transduced permanently? Gene delivery can also be used to study gene function in a variety of biological systems. The technology can create appropriate animal disease models. Gene delivery systems are being extensively used to identify the functions of novel genes—a great tool for functional genomics. NIGTR would also become a major player in sponsoring and perhaps even conducting clinical trials, a fertile ground for training the next generation of scientists capable of translating bench science to bedside. Technologies that can broadly influence the medicine we practice come along rarely. It is therefore wholly appropriate to set up an independent NIGTR to promote and support the myriad activities of gene therapy. Although many NIH institutes already support various aspects of gene therapy, a unified institute would lend focus and galvanize gene therapists. I think the NIGTR merits discussion before it is dismissed as a self-serving proposal from an interested party.