Globin gene therapy for treating hemoglobinopathies

• 收藏
• 分享

专利权人:

Memorial Sloan-Kettering Cancer Center

发明人:

SADELAIN, Michel,CABRIOLU, Annalisa

申请号：

AU2018378683

公开号：

AU2018378683A1

申请日:

2018.12.06

申请国别(地区):

AU

年份:

2020

代理人:

摘要：

The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.

来源网站：

中国工程科技知识中心

来源网址：

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