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Article

Utility of Self-Destructing CRISPR/Cas Constructs for Targeted Gene Editing in the Retina

2 Aug 2019

Abstract excerpt

Safe delivery of CRISPR/Cas endonucleases remains one of the major barriers to the widespread application of in vivo genome editing. 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 remain in the retina for an extended period, making genotoxicity a significant...

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PMID: 31373227
DOI: 10.1089/hum.2019.021

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