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Revolution in Gene Medicine Therapy and Genome Surgery

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Jonas Children’s Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory, Columbia University, New York, NY 10032, USA
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Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY 10032, USA
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Department of Pathology & Cell Biology, Stem Cell Initiative (CSCI), Institute of Human Nutrition, Vagelos College of Physicians and Surgeons, Columbia University, New York, NY 10032, USA
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Author to whom correspondence should be addressed.
Genes 2018, 9(12), 575; https://doi.org/10.3390/genes9120575
Received: 1 October 2018 / Revised: 17 November 2018 / Accepted: 20 November 2018 / Published: 26 November 2018
(This article belongs to the Section Human Genomics and Genetic Diseases)
Recently, there have been revolutions in the development of both gene medicine therapy and genome surgical treatments for inherited disorders. Much of this progress has been centered on hereditary retinal dystrophies, because the eye is an immune-privileged and anatomically ideal target. Gene therapy treatments, already demonstrated to be safe and efficacious in numerous clinical trials, are benefitting from the development of new viral vectors, such as dual and triple adeno-associated virus (AAV) vectors. CRISPR/Cas9, which revolutionized the field of gene editing, is being adapted into more precise “high fidelity” and catalytically dead variants. Newer CRISPR endonucleases, such as CjCas9 and Cas12a, are generating excitement in the field as well. Stem cell therapy has emerged as a promising alternative, allowing human embryo-derived stem cells and induced pluripotent stem cells to be edited precisely in vitro and then reintroduced into the body. This article highlights recent progress made in gene therapy and genome surgery for retinal disorders, and it provides an update on precision medicine Food and Drug Administration (FDA) treatment trials. View Full-Text
Keywords: gene therapy; gene editing; CRISPR/Cas9; Cas12a; dual AAV; triple AAV; clinical trials; retina; hereditary retinal dystrophies gene therapy; gene editing; CRISPR/Cas9; Cas12a; dual AAV; triple AAV; clinical trials; retina; hereditary retinal dystrophies
MDPI and ACS Style

Jiang, D.J.; Xu, C.L.; Tsang, S.H. Revolution in Gene Medicine Therapy and Genome Surgery. Genes 2018, 9, 575.

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