A multifunctional AAV–CRISPR–Cas9 and its host response

Wei Leong Chew,Mohammadsharif Tabebordbar,Jason K W Cheng,Prashant Mali,Elizabeth Y Wu,Alex H M Ng,Kexian Zhu,Amy J Wagers,George M Church

NATURE METHODS（2016）

引用 483|浏览33

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摘要

Packaging split Cas9 into AAVs increases cargo capacity and allows for efficient genome editing and gene activation in vivo . AAV–split-Cas9 activates the host immune system but does not trigger the extensive cellular damage observed with delivery of Cas9 via DNA electroporation.

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关键词

Preclinical research,Immunology,Gene targeting,Genetic engineering,Molecular engineering

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