TARGETING BCL11A DISTAL REGULATORY ELEMENTS FOR FETAL HEMOGLOBIN REINDUCTION

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专利权人:

The Children's Medical Center Corporation

发明人:

Stuart H. Orkin,Daniel E. Bauer,Jian Xu

申请号：

US16573389

公开号：

US20200087651A1

申请日:

2019.09.17

申请国别(地区):

US

年份:

2020

代理人:

摘要：

Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.

来源网站：

中国工程科技知识中心

来源网址：

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