Claim: Britain backs GSK's gene therapy for 'bubble boy' syndrome.
Verdict: SUPPORTS

Retrieved passages:
title: Jeans for Genes
text: Programme for care services for children and information and support for families. Funding from the appeal was key in the development of gene therapy at the UCL Institute of Child Health at Great Ormond Street Hospital in London, which has since cured ten children of the life-threatening condition X-linked severe combined immune deficiency X-SCID or 'baby in the bubble' syndrome. The charity also aims to raise awareness and understanding of genetics and what it means to live with a genetic disorder. It provides educational materials for schools through a dedicated website called Genes Are Us Jeans for Genes Jeans for

title: Gene therapy
text: treating children with SCID (severe combined immune deficiency or "bubble boy" disease) from 2000 and 2002, was questioned when two of the ten children treated at the trial's Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany. In 1993 Andrew Gobea was born with SCID following prenatal genetic screening. Blood was removed from his mother's placenta and umbilical cord immediately after birth, to acquire stem cells. The allele that codes for adenosine deaminase (ADA) was obtained and inserted

title: Duke University School of Medicine
text: children born with severe combined immunodeficiency, also known as bubble boy disease. 1984 Bart Haynes contributes to the identification of HTLV-III, now known as HIV. 1990 Joanne Kurtzberg establishes the Duke Pediatric Blood and Marrow Transplant Program. 1994 Dr. Louise Markert demonstrates that babies born with no immune system, a fatal condition known as complete DiGeorge syndrome, can be cured with thymus transplantation. 1995 Duke scientists link the BRCA1 and BRCA2 genes to breast and ovarian cancers. 2001 Miguel Nicolelis develops a system that allows monkeys to control robot arms via brain signals, an important step to enable paralyzed people

title: Gene therapy
text: infection, autoimmune diseases, and cancer. Follow up trials with gene therapy on another six children with Wiskott-Aldrich syndrome were also reported as promising. In October researchers reported that two children born with adenosine deaminase severe combined immunodeficiency disease (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and that their immune systems were showing signs of full recovery. Another three children were making progress. In 2014 a further 18 children with ADA-SCID were cured by gene therapy. ADA-SCID children have no functioning immune system and are sometimes known as "bubble children." Also in October researchers reported that

title: William French Anderson
text: William French Anderson William French Anderson (born December 31, 1936) is an American physician, geneticist and molecular biologist. He is considered a pioneer of gene therapy. He graduated from Harvard College in 1958 and from Harvard Medical School in 1963. In 1990, he claimed to be the first person ever to succeed in gene therapy of a 4-year-old girl suffering from severe combined immunodeficiency (a disorder called "bubble boy disease"). In 2006, he was convicted of sexual abuse of a minor and in 2007 was sentenced to 14 years in prison. Anderson was born in Tulsa, Oklahoma. His father was

title: Gene therapy
text: Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis and the European Commission approved it in June. This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene therapy treatment to be approved in Europe. In October, Chinese scientists reported they had started a trial to genetically modify T-cells from 10 adult patients with lung cancer and reinject the modified T-cells back into their bodies to attack the cancer cells. The T-cells had the PD-1 protein (which stops or slows the

title: Severe combined immunodeficiency
text: most severe form of primary immunodeficiencies, and there are now at least nine different known genes in which mutations lead to a form of SCID. It is also known as the bubble boy disease and bubble baby disease because its victims are extremely vulnerable to infectious diseases and some of them, such as David Vetter, have become famous for living in a sterile environment. SCID is the result of an immune system so highly compromised that it is considered almost absent. SCID patients are usually affected by severe bacterial, viral, or fungal infections early in life and often present with

title: William French Anderson
text: the 1980s. Prosecutors dropped those charges, citing insufficient evidence under Maryland law and credibility of the witness. Anderson was released from prison on May 18, 2018. William French Anderson William French Anderson (born December 31, 1936) is an American physician, geneticist and molecular biologist. He is considered a pioneer of gene therapy. He graduated from Harvard College in 1958 and from Harvard Medical School in 1963. In 1990, he claimed to be the first person ever to succeed in gene therapy of a 4-year-old girl suffering from severe combined immunodeficiency (a disorder called "bubble boy disease"). In 2006, he was

title: Gene therapy
text: levels of SERCA2, a protein in heart muscles, improving muscle function. The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process. In 2016 it was reported that no improvement was found from the CUPID 2 trial. In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 7–32 months. Three of the children had metachromatic leukodystrophy, which causes children to lose cognitive and motor skills. The other children had Wiskott-Aldrich syndrome, which leaves them to open to

title: Designer baby
text: Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, on a four-year-old girl named, Ashanti DeSilva, she was treated for ADA-SCID. This is a disease that had left her defenseless against infections spreading throughout her body. Dr. W French Anderson was a major lead on this clinical trial, he worked for the National Heart, Lung, and Blood Institute Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy. The techniques

title: Genetically modified organism
text: rising from adenosine deaminase deficiency (ADA-SCID), although the development of leukemia in some ADA-SCID patients along with the death of Jesse Gelsinger in another trial set back the development of this approach for many years. In 2009 another breakthrough was achieved when an eight year old boy with Leber’s congenital amaurosis regained normal eyesight and in 2016 GlaxoSmithKline gained approval to commercialise a gene therapy treatment for ADA-SCID. As of 2018, there are a substantial number of clinical trials underway, including treatments for hemophilia, glioblastoma, chronic granulomatous disease, cystic fibrosis and various cancers. The most common virus used for gene

title: Strimvelis
text: enzyme levels, anemia, neutropenia, hemolytic anaemia, aplastic anaemia and thrombocytopenia. The treatment was developed at San Raffaele Telethon Institute for Gene Therapy and developed by GlaxoSmithKline (GSK) through a 2010 collaboration with Fondazione Telethon and Ospedale San Raffaele. GSK, working with the biotechnology company MolMed S.p.A, developed a manufacturing process that was previously only suitable for clinical trials into one demonstrated to be robust and suitable for commercial supply. In April 2016, a committee at the European Medicines Agency recommended marketing approval for its use in children with adenosine deaminase deficiency, for whom no matched HSC donor is available, on

title: William French Anderson
text: Dr. Anderson wanted to test this theory with a human disease. In 1988 his proposal to the Human Gene Therapy Subcommittee was denied, however his request for a hearing before the full committee proceeded, and the trial was approved. In May 1989 he conducted the first human safety test for gene therapy, a harmless marker injected into a 53-year-old man. A year later a therapeutic trial was begun, to replace a defective ADA gene in a 4-year-old girl, called Ashanti DeSilva (also called Ashi). In 2007, at the age of 21, she was stable, but still had to take regular

title: Gene therapy
text: therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles. Some of the unsolved problems include: Three patients' deaths have been reported in gene therapy trials, putting the field under close scrutiny. The first was that of Jesse Gelsinger, who died in 1999 because of immune rejection response. One X-SCID patient died of leukemia in 2003. In 2007, a rheumatoid arthritis patient died from an infection; the subsequent investigation concluded that the death was not related to gene therapy. In 1972 Friedmann and Roblin authored a paper in

title: Carlos Pardo-Villamizar
text: as bubble boy disease." Pardo himself has stated, "We were concerned that the study would raise a lot of controversy and be misused. We were right." In addition, Pardo's team wrote an online primer accompanying the study's publication in which they stated that "THERE IS NO indication for using anti-inflammatory medications in patients with autism." He also noted, at the time of the study's publication, that "it is not yet clear whether the inflammation is a consequence of disease or a cause of it, or both," and that it was also not yet clear "whether it [immune activation] is destructive

title: 2002 French gene therapy trials
text: 2002 French gene therapy trials The 2002 French gene therapy trials were experimental gene therapy trials performed on children suffering from Severe Combined Immune Deficiency (SCID). The trials were based in Paris and lead by researchers Alain Fischer and Marina Cavazzana-Calvo. Whilst the experiment initially seemed successful, many of the children began showing symptoms of various cancer-like diseases as a result of the gene manipulation. The experiment, and others like it, were subsequently shut down. The experimental trials took place at the Necker Hospital in Paris, France in 2002. The lead researchers tasked with running the experiment were Dr. Alain

title: Kowarski syndrome
text: (normal, 14% to 39% in plasma) and the patients' growth hormone polymers were abnormally resistant to conversion into monomers by urea. Takahashi et al. reported a case of a boy with short stature who was heterozygous for a mutation in the GH1 gene. In this child, growth hormone not only could not activate the GH receptor (GHR) but also inhibited the action of wild type GH because of its greater affinity for GHR and GH-binding protein (GHBP) that is derived from the extracellular domain of the GHR. Thus, a dominant-negative effect was observed. Takahashi et al. demonstrated in a girl

title: Gene therapy
text: became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission. Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered – replacing or disrupting defective genes. Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase. DNA must be administered, reach the damaged cells, enter the cell and

title: 2002 French gene therapy trials
text: Fischer and Dr. Marina Cavazzana-Calvo, who were both employed by the hospital. The researchers were investigating treatments for Severe Combined Immune Deficiency, or SCID, a disease that had been linked to the X-chromosome. SCID has the effect of preventing the formation of several key immune system factors that aid in the body's ability to fight of infectious diseases. The goal of the gene therapy was to utilize and activate hematopoietic stem cells (HSCs) in the hopes of combating the progression of the immune deficiency. The methodology of the experiment involved using retroviral vectors to stimulate the HSCs, which had been

title: Gene therapy
text: gene of the patient's blood cells was replaced by the functional variant. Ashanti’s immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary. Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993). The treatment of glioblastoma multiforme, the malignant brain tumor whose outcome is always fatal, was done using a vector expressing antisense IGF-I RNA (clinical trial approved by NIH protocolno.1602 November