Gene editing of deep intronic mutations

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专利权人:

Genzyme Corporation

发明人:

Ruan, Guoxiang,Scaria, Abraham

申请号：

AU2016265255

公开号：

AU2016265255A1

申请日:

2016.04.15

申请国别(地区):

AU

年份:

2018

代理人:

摘要：

Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non- naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)— CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self- limiting CRISPER-Cas system.

来源网站：

中国工程科技知识中心

来源网址：

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