	US 12,391,947 B2
	Modulation of SPTLC1 via recombinant adeno-associated vectors
Christian Mueller, Worcester, MA (US); Gabriela Toro Cabrera, Worcester, MA (US); and Robert H. Brown, Jr., Worcester, MA (US)
Assigned to University of Massachusetts, Westborough, MA (US)
Appl. No. 17/297,521
Filed by University of Massachusetts, Boston, MA (US)
PCT Filed Nov. 27, 2019, PCT No. PCT/US2019/063558 § 371(c)(1), (2) Date May 27, 2021, PCT Pub. No. WO2020/112967, PCT Pub. Date Jun. 4, 2020.
Claims priority of provisional application 62/772,668, filed on Nov. 29, 2018.
Prior Publication US 2022/0033824 A1, Feb. 3, 2022
Int. Cl. C12N 15/113 (2010.01); A61K 35/761 (2015.01); C12N 15/10 (2006.01); C12N 15/86 (2006.01)

CPC C12N 15/1137 (2013.01) [A61K 35/761 (2013.01); C12N 15/102 (2013.01); C12N 15/86 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2750/14133 (2013.01); C12N 2750/14143 (2013.01); C12Y 203/0105 (2013.01)]

9 Claims

1. An isolated nucleic acid comprising a transgene encoding at least one inhibitory nucleic acid, wherein the at least one inhibitory nucleic acid comprises a nucleic acid sequence that is complementary to a nucleic acid encoding a serine palmitoyltransferase subunit, wherein the transgene is flanked by inverted terminal repeats (ITRs) derived from adeno-associated virus (AAV), wherein the at least one inhibitory nucleic acid is a microRNA (miRNA) or an artificial microRNA (amiRNA), and wherein the at least one inhibitory nucleic acid comprises:

(i) a first strand comprising SEQ ID NO: 16 and a second strand comprising SEQ ID NO: 17;

(ii) a first strand comprising SEQ ID NO: 18 and a second strand comprising SEQ ID NO: 19; or

(iii) a first strand comprising SEQ ID NO: 20 and a second strand comprising SEQ ID NO: 21.