Back to results
US 12,637,696 B2Granted
Augmentations to lentiviral vectors (CCLC-MGATA/ANK-CORE LCR-BETA-AS3-FB) to increase expression
AssigneeThe Regents of the University of California
InventorsDonald B. Kohn, Richard A. Morgan, Roger Paul Hollis
Life of this patent
Estimating the term…
Abstract
In certain embodiments a lentiviral vector having an LCR comprising HS1 ENCODE core (EC1) sequence (SEQ ID NO:1), and one or more of an HS2 core sequence (ecHS2), an HS3 core sequence (ecHS3), an HS4 core sequence (ecHS4), a full length HS2, a full length HS3, and/or a full length HS4 sequence is provided. In certain embodiments the vector comprises a modified βAS3-globin transgene, where transgene comprises a codon optimized exon 1, and/or a codon optimized exon 2, and/or a codon optimized exon 3.
Claims12
A recombinant lentiviral vector (LV) comprising:
- an expression cassette comprising a nucleic acid construct comprising:
- a human β-globin locus control region (LCR) comprising an HS1 ENCODE core (EC1) sequence set forth in SEQ ID NO:1, and a combination of an HS2 core sequence (ecHS2) set forth in SEQ ID NO:
- 2, an HS3 core sequence (ecHS3) set forth in SEQ ID NO:3, and an HS4 core sequence (ecHS4) set forth in SEQ ID NO:4, or a combination of a full length HS2, a full length HS3 and a full length HS4;
- and
- a heterologous gene to be expressed by the construct operably linked to the human β-globin LCR.
2 more elements
Dependencies are inferred from the claim text.