US 12,281,304 B2
Compositions and methods for treating and preventing muscular disorders and dystrophies, steroid side effects, and inflammation
Christopher Heier, Washington, DC (US); and Alyson Fiorillo, Washington, DC (US)
Assigned to Children's National Medical Center, Washington, DC (US)
Appl. No. 17/059,700
Filed by Children's National Medical Center, Washington, DC (US)
PCT Filed Jun. 3, 2019, PCT No. PCT/US2019/035264
§ 371(c)(1), (2) Date Nov. 30, 2020,
PCT Pub. No. WO2019/232548, PCT Pub. Date Dec. 5, 2019.
Claims priority of provisional application 62/679,529, filed on Jun. 1, 2018.
Prior Publication US 2021/0198669 A1, Jul. 1, 2021
Int. Cl. C12N 15/113 (2010.01); A61K 31/573 (2006.01); A61K 31/7088 (2006.01); A61P 21/00 (2006.01); C12Q 1/68 (2018.01)
CPC C12N 15/113 (2013.01) [A61K 31/573 (2013.01); A61K 31/7088 (2013.01); A61P 21/00 (2018.01); C12N 2310/11 (2013.01); C12N 2320/31 (2013.01); C12N 2320/35 (2013.01)] 24 Claims
 
1. A method of treating a muscle disease in a subject in need thereof, the method comprising administering one or more inhibitory antisense oligonucleotide (ASO) molecules or one or more expression vectors encoding the one or more inhibitory ASO molecules, the one or more inhibitory antisense oligonucleotide molecules inhibit or reduce activity of at least one NF kappa B regulated micro RNA (miRNA) molecules in an amount sufficient to reduce one or more symptoms of the muscle disease,
wherein the ASO molecule is complementary to one or a combination of miR-142-3p (SEQ ID NO:12), miR-142-5p (SEQ ID NO:13), miR-652 (SEQ ID NO:19), miR-455-3p (SEQ ID NO:6), and miR-497-5p (SEQ ID NO:7) or at least 70% complementary to miR-142-3p (SEQ ID NO:12), miR-142-5p (SEQ ID NO:13), miR-652 (SEQ ID NO:19), miR-455-3p (SEQ ID NO:6), and miR-497-5p (SEQ ID NO:7).