Donald B. Kohn,Fabrizia Urbinati,Zulema R. Garcia,Roger P. Hollis,Sabine Geiger-Schredelseker,Aaron R. Cooper,Shantha Senadheera
申请号：
US15347657
公开号：
US20170157270A1
申请日:
2016.11.09
申请国别(地区):
US
年份:
2017
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.
