Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina.
Methods in molecular biology (Clifton, N.J.), 1950: 123-139 PMID 30783971 DOI 10.1007/978-1-4939-9139-6_7
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.