bluebird bio, Inc.;Massachusetts Institute of Technology
发明人:
Leboulch Philippe Louis,Pawliuk Robert,Westerman Karen
申请号：
US201514700021
公开号：
US10105451(B2)
申请日:
2015.04.29
申请国别(地区):
美国
年份:
2018
代理人:
Cooley LLP
摘要：
The invention provides improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. The invention further provides improved gene therapy methods for treating hematopoietic-related disorders. Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.
