深內含子突變的基因編輯

• 收藏
• 分享

专利权人:

Genzyme Corporation

发明人:

RUAN, Guoxiang,SCARIA, Abraham

申请号：

HK18111777

公开号：

HK1252469A1

申请日:

2018.09.13

申请国别(地区):

HK

年份:

2019

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/