References

1. Wirth T, Parker N, Yl-Hertuala. History of gene therapy. Gene. 2013;252(2):62-169.2. Food and Drug Administration. FDA continues strong support of innovation in development of gene therapy products. Press release. Accessed July 1, 2021. https://www.fda.gov/news-events/press-announcements/fda-continues-strong-support-innovation-development-gene-therapy-products3. Science History Institute. James Watson, Francis Crick, Maurice Wilkins, and Rosalind Franklin. Accessed July 1, 2021. https://www.sciencehistory.org/historical-profile/james-watson-francis-crick-maurice-wilkins-and-rosalind-franklin4. Nirenberg M. Historical review: Deciphering the genetic codea personal account. Trends Biochem Sci. 2004;29(1):46-54.5. Science History Institute. Herbert W Boyer and Stanley N Cohen. Accessed July 1, 2021. https://www.sciencehistory.org/historical-profile/herbert-w-boyer-and-stanley-n-cohen6. Sun M. Cline loses two NIH grants. Science. 1981;214(4525):1220.7. Blaese RM, Culver KW, Miller D, et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science. 1995;270(5235):475-480.8. Kim YG, Cha J, Chandrasegaran S. Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain. Proc Natl Acad Sci U S A. 1996;93(3):1156-1160.9. Naldini L, Blomer U, Gallay P, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 1996;272(5259):263-267.10. Sibbald B. Death but one unintended consequence of gene-therapy trial. CMAJ. 2001;164(11):1612.11. Hacein-Bey-Abina S, Garrigue A, Wang GP, et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest. 2018;118(9):3132-3142.12. Cavazzana-Calvo M, Fischer A. Gene therapy for severe combined immunodeficiency: are we there yet? J Clin Invest. 2007;117(6):1456-1465.13. Humeau L. From the bench to the clinic: story and lessons from VRX496, the first lentivector ever tested in a phase 1 clinical trial. Presented at: Beilstein Bozen Symposium; May 15-May 19, 2006; Bozen, Italy.14. Pearson S, Jia H, Kandachi K. China approves first gene therapy. Nat Biotechnol. 2004;22(1):3-4. 15. Daley J. Gene therapy arrives. Nature. 2019;576:S12-S13.16. Maguire AM, High KA, Auricchio A, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet. 2009;374(9701):1597-1605.17. Luxturna (voretigene neparvovec-ryzl) [prescribing information]. Philadelphia, PA: Spark Therapeutics, Inc.; 2017.18. Christian M, Cermak T, Doyle EL, et al. Targeting DNA double-strand breaks with TAL effector nucleases. Genetics. 2010;186(2):757-761.19. Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human -thalassaemia. Nature. 2010;467(7313):318-322.20. Flemming A. Regulatory watch: Pioneering gene therapy on brink of approval. Nat Rev Drug Discov. 2012 ;11(9):664.21. Pharmaphorum. Glybera, the most expensive drug in the world, to be withdrawn after commercial flop. Accessed April 29, 2021. https://pharmaphorum.com/news/glybera-expensive-drug-world-withdrawn-commercial-flop/22. Cong L, Ran FA, Cox D, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339(6121):819-823.23. Aiuti A, Roncarolo MG, Naldini L. Gene therapy for ADA-SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products. EMBO Mol Med. 2017;9(6):737-740.24. Strimvelis Summary of Product Characteristics, GlaxoSmithKline (GSK); 2016.25. Food and Drug Administration. FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma. Accessed April 27, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-car-t-cell-therapy-treat-adults-certain-types-large-b-cell-lymphoma26. European Medicines Agency. Yescarta. Accessed April 29, 2021. https://www.ema.europa.eu/en/medicines/human/EPAR/yescarta27. Cross R. CRISPR is coming to the clinic this year. Chem Eng News. 2018;96(2):18-19.28. Food and Drug Administration. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. Accessed April 27, 2021. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease29. European Medicines Agency. Zolgensma. Accessed May 26, 2021. https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma30. European Medicines Agency. Zynteglo. Accessed April 29, 2021. https://www.ema.europa.eu/en/medicines/human/referrals/zynteglo31. Regulatory Affairs Professional Society. FDA finalizes 6 gene therapy guidances, unveils a new draft. Accessed April 27, 2021. https://www.raps.org/news-and-articles/news-articles/2020/1/fda-finalizes-6-gene-therapy-guidances-unveils-a 32. PR Newswire. 4-day-old baby receives life-changing $2M gene therapy at woman's hospital in Baton Rouge. Accessed April 27, 2021. https://www.prnewswire.com/news-releases/4-day-old-baby-receives-life-changing-2m-gene-therapy-at-womans-hospital-in-baton-rouge-301233580.html

Visit link:

History of Gene Therapy | Discovery and Evolution