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Article

Efficacy and dynamics of self-targeting CRISPR/Cas constructs for gene editing in the retina

2018-01-05

Abstract excerpt

<h4>ABSTRACT</h4> Safe delivery of CRISPR/Cas endonucleases remains one of the major barriers to the widespread application of in vivo genome editing including the anticipatory treatment of monogenic retinal diseases. We previously reported the utility of adeno-associated virus (AAV)-mediated CRISPR/Cas genome editing in the retina; however, with this type of viral delivery system, active endonucleases will rema...

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Literature Corpus work
b3821e42-fb11-5748-844e-7f5fc9369b25
DOI
10.1101/243683
Open publication

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