US 12,060,566 B2
Self-inactivating lentiviral vector comprising a FOXP3 expression cassette
Donald B. Kohn, Tarzana, CA (US); Maria Grazia Roncarolo, Menlo Park, CA (US); Roger Paul Hollis, Los Angeles, CA (US); Katelyn E Masiuk, Santa Monica, CA (US); and Rosa Bacchetta, Menlo Park, CA (US)
Assigned to THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, Oakland, CA (US); and THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY, Stanford, CA (US)
Appl. No. 16/640,306
Filed by The Regents of the University of California, Oakland, CA (US); and The Board of Trustees of the Leland Stanford Junior University, Stanford, CA (US)
PCT Filed Aug. 22, 2018, PCT No. PCT/US2018/047586
§ 371(c)(1), (2) Date Feb. 19, 2020,
PCT Pub. No. WO2019/040655, PCT Pub. Date Feb. 28, 2019.
Claims priority of provisional application 62/548,891, filed on Aug. 22, 2017.
Prior Publication US 2020/0347404 A1, Nov. 5, 2020
Int. Cl. C12N 15/86 (2006.01); C12N 5/0789 (2010.01)
CPC C12N 15/86 (2013.01) [C12N 5/0647 (2013.01); C12N 2501/60 (2013.01); C12N 2740/16043 (2013.01)] 13 Claims
OG exemplary drawing
 
1. 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 human FoxP3 promoter;
where said expression cassette comprises enhancer elements said enhancer elements consisting of a human FoxP3-CNS1 sequence, a human FoxP3-CNS2 sequence, and a human FoxP3-CNS3 sequence upstream from said 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 U5 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 a human FoxP3 protein; and
where said LV has a length of less than 10 kb.