COOPER, AARON,KOHN, DONALD B.,URBINATI, FABRIZIA,GARCIA, ZULEMA R.,HOLLIS, ROGER,GEIGER-SCHREDELSEKER, SABINE,SENADHEERA, SHANTHA
申请号：
EP13836738
公开号：
EP2895611A4
申请日:
2013.09.10
申请国别(地区):
EP
年份:
2016
代理人:
摘要：
In various embodiments a recombinant lentiviral vector is provided comprising an expression cassette comprising a nucleic acid construct comprising an anti-sickling human beta globin gene encoding an anti-sickling-beta globin polypeptide comprising the mutations Gly16Asp, Glu22Ala and Thr87Gln, where the lentiviral vector is a TAT-independent and self-inactivating (SIN). In certain embodiments the vector additionally contains one or more insulator elements. The vectors are useful in gene therapy for the treatment of sickle cell disease.
