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US 12,747,454 B2Granted
Self-inactivating lentiviral vector comprising a FoxP3 expression cassette
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.
Claims10
A recombinant lentiviral vector (LV) comprising:
- an expression cassette comprising enhancer elements for driving gene expression of a nucleic acid encoding a protein, said enhancer elements consisting of a human FoxP3-CNS1 sequence, a human FoxP3-CNS2 sequence, and a human FoxP3-CNS3 sequence upstream from a human FoxP3 promoter;
- where said LV is an HIV-derived TAT-independent and self-inactivating (SIN) lentiviral vector comprising a U3 region lacking a TATA box, an R region, a U 5 region, a packaging signal, a rev response element (RRE), and a central polypurine tract (cPPT) upstream from said enhancer elements, U3 region, R region, and U5 region downstream from said nucleotide sequence encoding the protein;
- and
- a nucleic acid sequence encoding said protein sequence operably linked to said enhancer elements and said human FoxP3 promoter.
1 more element
Dependencies are inferred from the claim text.