GENE EDITING OF DEEP INTRONIC MUTATIONS

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专利权人:

发明人:

RUAN GUOXIANG,SCARIA ABRAHAM

申请号：

IN201717045050

公开号：

IN201717045050A

申请日:

2017.12.14

申请国别(地区):

IN

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/