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

Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina.
Methods Mol Biol. 2019;1950:123-139
Authors: Yu W, Wu Z
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.
PMID: 30783971 [PubMed - indexed for MEDLINE]

Source: Mol Biol Cell - July 17, 2019 Category: Molecular Biology Authors: Yu W, Wu Z Tags: Methods Mol Biol Source Type: research