ADMINISTRATION D'AAV D'EDITEURS DE NUCLEOBASES

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专利权人:

PRESIDENT AND FELLOWS OF HARVARD COLLEGE

发明人:

LIU, DAVID R.,LEVY, JONATHAN MA,YEH, WEI HSI

申请号：

CA3039928

公开号：

CA3039928A1

申请日:

2017.10.13

申请国别(地区):

CA

年份:

2018

代理人:

摘要：

Provided herein are methods of delivering "split" Cas9 protein or nucleobase editors into a cell, e.g., via a recombinant adeno-associated virus (rAAV), to form a complete and functional Cas9 protein or nucleobase editor. The Cas9 protein or the nucleobase editor is split into two sections, each fused with one part of an intein system (e.g., intein-N and intein-C encoded by dnaEn and dnaEc, respectively). Upon co-expression, the two sections of the Cas9 protein or nucleobase editor are ligated together via intein-mediated protein splicing. Recombinant AAV vectors and particles for the delivery of the split Cas9 protein or nucleobase editor, and methods of using such AAV vectors and particles are also provided.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/