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US 2020/0347404 A1Published application
Lentiviral vectors expressing foxp3 in hematopoietic stem cells to treat immune deficiencies and autoimmune diseases
AssigneesThe Regents of the University of California, The Board of Trustees of the Leland Stanford Junior University
InventorsDonald B. Kohn, Maria Grazia Roncarolo, Roger Paul Hollis, Katelyn E. Masiuk, Rosa Bacchetta
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Abstract
In various embodiments, lentiviral vectors expressing FoxP3 using the endogenous gene elements to regulate physiologic gene expression of FoxP3 are provided herein as well as uses of such vectors.
Claims24
A recombinant lentiviral vector (LV) comprising:
- an expression cassette comprising a nucleic acid construct comprising a nucleotide sequence encoding a human FoxP3 protein operably linked to an endogenous FoxP3 promoter;
- where said LV is a TAT-independent and self-inactivating (SIN) lentiviral vector.
9-16. (canceled)
23-28. (canceled)
An infectious lentivirus particle comprising a nucleic acid that expresses a human FoxP3 protein.
36-41. (canceled)
Dependencies are inferred from the claim text.