MEMORIAL SLOAN-KETTERING CANCER CENTER;UNIVERSITY OF WASHINGTON
发明人:
Michel Sadelain,Isabelle Riviere,Jorge Mansilla-Soto,Xiuyan Wang,George Stamatoyannopoulos,John Stamatoyannopoulos,Mingdong Liu
申请号：
US15449416
公开号：
US20170173185A1
申请日:
2017.03.03
申请国别(地区):
US
年份:
2017
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.
