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.
CITATION STYLE
Yu, W., & Wu, Z. (2019). Use of AAV vectors for CRISPR-mediated in vivo genome editing in the retina. In Methods in Molecular Biology (Vol. 1950, pp. 123–139). Humana Press Inc. https://doi.org/10.1007/978-1-4939-9139-6_7
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