	US 11,883,470 B2
	Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
Daniel Rader, Philadelphia, PA (US); Devin Christopher, Philadelphia, PA (US); and Anna P. Tretiakova, Philadelphia, PA (US)
Assigned to The Trustees of the University of Pennsylvania, Philadelphia, PA (US)
Appl. No. 16/320,350
Filed by The Trustees of the University of Pennsylvania, Philadelphia, PA (US)
PCT Filed Jul. 24, 2017, PCT No. PCT/US2017/043535 § 371(c)(1), (2) Date Jan. 24, 2019, PCT Pub. No. WO2018/022511, PCT Pub. Date Feb. 1, 2018.
Claims priority of provisional application 62/366,423, filed on Jul. 25, 2016.
Prior Publication US 2019/0282670 A1, Sep. 19, 2019
Int. Cl. A61K 38/45 (2006.01); A61K 48/00 (2006.01); C12N 9/10 (2006.01); C12N 15/86 (2006.01); A61P 9/00 (2006.01); C12P 21/02 (2006.01); A61P 1/16 (2006.01); A61K 38/00 (2006.01)

CPC A61K 38/45 (2013.01) [A61K 48/0058 (2013.01); A61P 1/16 (2018.01); A61P 9/00 (2018.01); C12N 9/1029 (2013.01); C12N 15/86 (2013.01); C12P 21/02 (2013.01); A61K 38/00 (2013.01); C12N 2750/14143 (2013.01); C12Y 203/01043 (2013.01)]

25 Claims

1. A recombinant viral vector which comprises an expression cassette comprising:

(a) a nucleic acid sequence comprising SEQ ID NO: 26, SEQ ID NO: 63, or a sequence at least 95% identical to SEQ ID NO: 26 or 63 which encodes a functional human lecithin cholesterol acyltransferase (LCAT) variant comprising: an M amino acid substitution at amino acid residue position 114 based on the residue numbering of WT normal LCAT (V114M); and

(b) regulatory sequences which direct expression of the LCAT V114M in a host cell, said regulatory sequences being operably linked to the LCAT V114M sequence.