	US 11,976,293 B2
	Optimized lentiviral vector for stem cell gene therapy of hemoglobinopathies
Donald B. Kohn, Tarzana, CA (US); Richard A. Morgan, Los Angeles, CA (US); and Roger P. Hollis, Los Angeles, CA (US)
Assigned to THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, Oakland, CA (US)
Appl. No. 16/466,970
Filed by The Regents of the University of California, Oakland, CA (US)
PCT Filed Dec. 5, 2017, PCT No. PCT/US2017/064766 § 371(c)(1), (2) Date Jun. 5, 2019, PCT Pub. No. WO2018/106724, PCT Pub. Date Jun. 14, 2018.
Claims priority of provisional application 62/430,157, filed on Dec. 5, 2016.
Prior Publication US 2020/0109416 A1, Apr. 9, 2020
Int. Cl. C12N 15/86 (2006.01); A61K 35/28 (2015.01); A61K 48/00 (2006.01); A61P 7/00 (2006.01); C12N 7/00 (2006.01)

CPC C12N 15/86 (2013.01) [A61K 35/28 (2013.01); A61K 48/005 (2013.01); A61P 7/00 (2018.01); C12N 7/00 (2013.01); C12N 2740/15043 (2013.01)]

12 Claims

1. A recombinant lentiviral vector (LV) comprising:

an expression cassette comprising a nucleic acid construct comprising:

a human β-globin locus control region consisting of a hypersensitive site (HS) core sequence consisting of:

an HS2 core sequence that consists of a nucleic acid sequence ranging from nucleotide 5484 to 5908 of SEO ID NO: 1;

an HS3 core sequence that consists of a nucleic acid sequence ranging from nucleotide 5909 to 6252 of SEO ID NO: 1; and

an HS4 core sequence that consists of a nucleic acid sequence ranging from nucleotide 6253 to 6662 of SEO ID NO: 1;

and a recombinant human beta globin gene encoding a beta globin polypeptide, wherein said LV is a TAT-independent and self-inactivating (SIN) lentiviral vector.