Memorial Sloan-Kettering Cancer Center;University of Washington
发明人:
申请号：
EP19185730.9
公开号：
EP3620521A1
申请日:
2015.09.04
申请国别(地区):
EP
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.
