[PERSPECTIVES] Therapeutic Gene Editing in Inherited Retinal Disorders

Jinjie Ling1, Laura A. Jenny2, Ashley Zhou1 and Stephen H. Tsang2,3,4,5 1Columbia University Vagelos College of Physicians and Surgeons, New York, New York 10032, USA 2Jonas Children's Vision Care, and Bernard and Shirley Brown Glaucoma Laboratory, Edward Harkness Eye Institute, Department of Ophthalmology, New York-Presbyterian Hospital, New York, New York 10032, USA 3Department of Biomedical Engineering, Columbia University, New York, New York 10032, USA 4Columbia Stem Cell Initiative, and Institute of Human Nutrition, Columbia University, New York, New York 10032, USA 5Department of Pathology and Cell Biology, Columbia University, New York, New York 10032, USA Correspondence: sht2columbia.edu

Since the development of CRISPR/Cas9 gene editing in 2012, therapeutic editing research has produced several phase 1-2a trials. Here we provide an overview of the mechanisms and applications of various gene-editing technologies including adeno-associated virus vectors, lentiviruses, CRISPR/Cas9 systems, base and prime editing, antisense oligonucleotides, short-hairpin RNAs, Cas13, and adenosine deaminase acting on RNA for the treatment of various inherited retinal diseases (IRDs). We outline the various stages of clinical trials using these technologies and the impacts they have made in advancing the practice of medicine.

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