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LENTIVIRAL VECTOR FOR STEM CELL GENE THERAPY OF SICKLE CELL DISEASE
专利权人:
发明人:
申请号:
EP13836738.8
公开号:
EP2895611A1
申请日:
2013.09.10
申请国别(地区):
EP
年份:
2015
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.
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