Article
The Application of CRISPR/Cas9 for the Treatment of Retinal Diseases.
The Yale journal of biology and medicine - 1 Dec 2017
Peddle Caroline F, MacLaren Robert E
Abstract excerpt
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....
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