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. 2019:1950:123-139.
doi: 10.1007/978-1-4939-9139-6_7.

Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina

Wenhan Yu  1 , Zhijian Wu  2
Affiliations

Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina

Wenhan Yu et al. Methods Mol Biol. 2019.

Abstract

Degenerative retinal diseases such as retinitis pigmentosa (RP) and Leber's congenital amaurosis (LCA) may lead to blindness without effective treatment. With the rapid advancement of the CRISPR/Cas9 genome editing technology, in vivo application of CRISPR/Cas9 holds immense potential for treatment of these diseases. Adeno-associated virus (AAV) vectors are an ideal gene transfer tool for delivery of CRISPR components to the retina. Here, we describe a protocol for utilizing an AAV-based CRISPR/Cas9 system for in vivo genome editing in the retina.

Keywords: AAV vector; CRISPR/Cas9; Gene therapy; Genome editing; Retina; Retinal degeneration.

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Figures

Fig. 1
Fig. 1
Sample SURVEYOR assay result. The SURVEYOR nuclease recognizes the mismatch (if any) in the reannealed heteroduplex, which results from indels introduced into the predicted cleavage site in the CRISPR edited cells. Black arrow: undigested amplicons; black triangles: cleavage products of the SURVEYOR nuclease
See this image and copyright information in PMC

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