BLUEBIRD BIO, INC.;MASSACHUSETTS INSTITUTE OF TECHNOLOGY
发明人:
Philippe Louis Leboulch,Robert Pawliuk,Karen Westerman
申请号：
US14700021
公开号：
US10105451B2
申请日:
2015.04.29
申请国别(地区):
US
年份:
2018
代理人:
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.
