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US 12,758,653 B2Granted
Lentiviral vectors in hematopoietic stem cells to treat X-linked chronic granulomatous disease
AssigneeThe Regents of the University of California
InventorsDonald B. Kohn, Ryan L. Wong, Roger Paul Hollis
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Abstract
Described herein is a lentiviral vector (LV) for the treatment of X-linked chronic granulomatous disease (X-CGD). The vector comprises an expression cassette comprising a nucleic acid construct comprising a CYBB promoter or effective fragment thereof, and a nucleic acid that encodes gp91phox operably linked to the CYBB promoter or promoter fragment thereof.
Claims12
A recombinant lentiviral vector (LV) comprising:
- an expression cassette comprising a nucleic acid construct comprising:
- a CYBB promoter or effective fragment thereof;
- and
- a nucleic acid that encodes a Gp91phox operably linked to said CYBB promoter or promoter fragment thereof;
- wherein said CYBB promoter or effective fragment thereof comprises the nucleic acid sequence of SEQ ID NO:
- 3;
- and
- wherein said expression cassette further comprises regulatory nucleic acid sequences comprising enhancers consisting of SEQ ID NO:
- 6 and SEQ ID NO:
- 11 for expression of the nucleic acid that encodes the Gp91phox operably linked to said CYBB promoter.
7 more elements
A recombinant lentiviral vector (LV) comprising SEQ ID NO:
- 22.
Dependencies are inferred from the claim text.