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The CRISPR/Cas9 system of genome editing has revolutionized molecular biology, offering a simple, and relatively inexpensive method of creating precise DNA edits. It has potential application in gene therapy treatment of retinal diseases providing targeted disruption, alteration, or transcriptional regulation of pathogenic genes. In vivo studies have demonstrated therapeutic benefit for a variety of diseases. Despite this, there are many challenges to clinical use of CRISPR/Cas9, including editing efficiency, off-target effects, and disease heterogeneity. This review details the mechanisms of the CRISPR/Cas9 system and the treatment strategies that can be applied to retinal diseases. It gives an overview of in vivo studies published to date and discusses the challenges and potential solutions to the wide-scale clinical use of CRISPR/Cas9 as a therapeutic intervention.

Type

Journal article

Journal

Yale J Biol Med

Publication Date

12/2017

Volume

90

Pages

533 - 541

Keywords

CRISPR, Cas9, HDR, NHEJ, gene editing, gene therapy, retinal disease, sgRNA, CRISPR-Cas Systems, Gene Editing, Gene Silencing, Genetic Therapy, Humans, Mutation, Retinal Diseases, Stem Cells