Article

Precision Cas9 Genome Editing <i>in vivo</i> with All-in-one, Self-targeting AAV Vectors

2020-10-09

Abstract excerpt

Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but are severely constrained by cargo limits, especially for large effectors like Cas9s. Simultaneous delivery of multiple vectors can limit dose and efficacy and increase safety risks. The use of compact effectors has enabled single-AAV delivery of Cas9s with 1-3 guides for edits that use end-joining repair pathwa...

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Literature Corpus work: c5a47948-a25a-5c04-9e76-32bf13f5969d
DOI: 10.1101/2020.10.09.333997

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