Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Revolution in Gene Medicine Therapy and Genome Therapy

Version 1 : Received: 1 October 2018 / Approved: 1 October 2018 / Online: 1 October 2018 (13:52:23 CEST)

A peer-reviewed article of this Preprint also exists.

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


Recently, there have been revolutions in the development of both gene therapy and genome surgical treatments for inherited diseases. Much of this progress has been centered around 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 AAVs. CRISPR/Ca9, which revolutionized the field of gene editing, is being adapted into more precise “high fidelity” and catalytically dead variants. New 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 FDA treatment trials.


Gene therapy, gene editing, CRISPR/Cas9, Cas12a, dual AAV, triple AAV, clinical trials, retina, hereditary retinal dystrophies


Medicine and Pharmacology, Ophthalmology

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