EDITION GENIQUE DE MUTATIONS INTRONIQUES PROFONDES

• 收藏
• 分享

专利权人:

GENZYME CORPORATION

发明人:

RUAN, GUOXIANG,SCARIA, ABRAHAM

申请号：

CA2986021

公开号：

CA2986021A1

申请日:

2016.04.15

申请国别(地区):

CA

年份:

2016

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/