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US 12,714,732 B2Granted
Lentiviral vectors in hematopoietic stem cells to treat Wiskott-Aldrich syndrome (WAS)
AssigneeThe Regents of the University of California
InventorsDonald B. Kohn, Ryan L. Wong, Roger Paul Hollis
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Abstract
In certain embodiments a lentiviral vector for the treatment of Wiskott-Aldrich Syndrome (WAS) is provided. In certain embodiments the vector comprises an expression cassette comprising a nucleic acid construct comprising an effective fragment of the endogenous promoter of the WAS gene where said promoter has maximum length of 600 bp and contains the sequence of HS1pro, and a nucleic acid that encodes the Wiskott-Aldrich Syndrome protein (WASp) operably linked to the effective fragment of the endogenous promoter of the WAS gene.
Claims11
A recombinant nucleic acid comprising:
- a nucleic acid sequence comprising or consisting of the nucleotide sequence of SEQ ID NO:
- 1 operably linked to a nucleic acid sequence comprising or consisting of the nucleotide sequence of SEQ ID NO:2 or a fragment thereof wherein fragment thereof comprises one of the following:
- the nucleic acid sequence comprising SEQ ID NO:3;
- the nucleic acid sequence comprising SEQ ID NO:4;
- the nucleic acid sequence comprising SEQ ID NO:5;
- the nucleic acid sequence comprising SEQ ID NO:6;
- the nucleic acid sequence comprising SEQ ID NO:7;
- or
- the nucleic acid sequence comprising SEQ ID NO:8.
6 more elements
Dependencies are inferred from the claim text.