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


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.

SUBMITTER: Yu W 

PROVIDER: S-EPMC6730636 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature

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