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Article

Comparison of CRISPR/Cas endonucleases for <i>in vivo</i> retinal gene editing

2020-06-09

Abstract excerpt

<h4>ABSTRACT</h4> CRISPR/Cas has opened the prospect of direct gene correction therapy for some inherited retinal diseases. Previous work has demonstrated the utility of adeno-associated virus (AAV) mediated delivery to retinal cells in vivo ; however, with the expanding repertoire of CRISPR/Cas endonucleases, it is not clear which of these are most efficacious for retinal editing in vivo . We sought to compare...

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Literature Corpus work
0a8e7f46-7b2b-5ca5-a839-5c159a386e9f
DOI
10.1101/2020.06.09.141705
Open publication

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