THERAPIE GENIQUE AVEC UN GENE DE GLOBINE POUR LE TRAITEMENT DES HEMOGLOBINOPATHIES

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专利权人:

MEMORIAL SLOAN-KETTERING CANCER CENTER

发明人:

SADELAIN, MICHEL,CABRIOLU, ANNALISA

申请号：

CA3084186

公开号：

CA3084186A1

申请日:

2018.12.06

申请国别(地区):

CA

年份:

2019

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/