Development of a Web Course on Gene Therapy by the International Consortium of Gene Therapy

To the editor: At the May 2013 meeting of the American Society of Gene & Cell Therapy in Salt Lake City, Utah, a group of researchers founded the International Consortium of Gene Therapy (ICGT). The mandate of this consortium is to lobby governments around the world to increase research funding for gene therapy, which has the potential to treat many hereditary and acquired diseases. Indeed, successful clinical gene therapy results have been obtained in recent years. Among these, it is worth noting the improvements in efficacy and safety demonstrated in new hematopoietic clinical trials conducted with lentiviral vectors,1Cavazzana-Calvo M Payen E Negre O Wang G Hehir K Fusil F et al.Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.Nature. 2010; 467: 318-322Crossref PubMed Scopus (1035) Google Scholar,2Biffi A Montini E Lorioli L Cesani M Fumagalli F Plati T et al.Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.Science. 2013; 341: 1233158Crossref PubMed Scopus (906) Google Scholar,3Aiuti A Biasco L Scaramuzza S Ferrua F Cicalese MP Baricordi C et al.Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.Science. 2013; 341: 1233151Crossref PubMed Scopus (809) Google Scholar as well as the progress in phase I clinical trials in hemophilia patients with an adeno-associated virus (AAV) vector carrying the clotting factor IX gene.4Nathwani AC Tuddenham EG Rangarajan S Rosales C McIntosh J Linch DC et al.Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.N Engl J Med. 2011; 365: 2357-2365Crossref PubMed Scopus (1354) Google Scholar In addition, an AAV vector has been used successfully to treat the hereditary blindness Leber's congenital amaurosis. Finally, Glybera has been approved for commercialization in Europe—the first gene therapy product to be approved—for patients with familial lipoprotein lipase deficiency.5Stein EA Dufour R Gagne C Gaudet D East C Donovan JM et al.Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebo-controlled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease.Circulation. 2012; 126: 2283-2292Crossref PubMed Scopus (249) Google Scholar With the recent progress in the development of specific endonucleases (transcription activator-like effector nucleases, zinc-finger nucleases, meganucleases, and, more recently, CRISPR/Cas9) to target the genome to correct or knock out specific genes,6Mali P Yang L Esvelt KM Aach J Guell M DiCarlo JE et al.RNA-guided human genome engineering via Cas9.Science. 2013; 339: 823-826Crossref PubMed Scopus (6424) Google Scholar,7Cermak T Doyle EL Christian M Wang L Zhang Y Schmidt C et al.Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting.Nucleic Acids Res. 2011; 39: e82Crossref PubMed Scopus (1560) Google Scholar,8Gaj T Gersbach CA Barbas CF ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.Trends Biotechnol. 2013; 31 (3rd): 397-405Abstract Full Text Full Text PDF PubMed Scopus (2481) Google Scholar,9Maeder ML Linder SJ Cascio VM Fu Y Ho QH Joung JK CRISPR RNA-guided activation of endogenous human genes.Nat Methods. 2013; 10: 977-979Crossref PubMed Scopus (800) Google Scholar it can be anticipated that these successes are only the beginning. Outside the gene therapy research community, however, the potential of these gene therapy approaches is not well known or understood. In fact, gene therapy is rarely included in the syllabus of most undergraduate courses and even less frequently in medical school studies. However, if we want governments to increase research funding for gene therapy and to interest young scientists and clinicians in working in the field, it is important to harvest the support of the larger biomedical community. To fulfill that goal, several ICGT members have created the basis of a Web course on gene therapy (http://www.internationalconsortiumofgenetherapy.com/course) comprising PowerPoint presentations contributed by global experts in various aspects of gene therapy. The presentations can be freely downloaded from the website and are thus accessible to the wider public, including the media and biology and medical students and their professors, who may use these documents to prepare their own lectures. The Web course, still in its infancy, thus far includes presentations from 17 researchers. Contributions from other experts in gene therapy are certainly encouraged. Topics of interest are indicated on the website, many of which lack contributions at this stage; potential contributors should contact us at [email protected] Suggestions for additional topics are also welcomed. Because PowerPoint presentations are typically too large to attach to e-mail messages, these may be submitted using WeTransfer (https://www.wetransfer.com). A review committee to be formed by the executive committee of the ICGT will review all new contributions to ensure that they are of high quality and up-to-date. The URL for the course will be posted on the websites of several associations related to gene and cell therapy.

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