	US 12,144,822 B2
	Mir-17˜92 as therapeutic or diagnostic target of motor neuron (MN) degeneration diseases
Jun-An Chen, Taipei (TW); Kuan-Chih Peng, Taipei (TW); and Ying-Tsen Tung, Taipei (TW)
Assigned to ACADEMIA SINICA, Taipei (TW)
Appl. No. 17/043,891
Filed by Academia Sinica, Taipei (TW)
PCT Filed Apr. 2, 2019, PCT No. PCT/US2019/025406 § 371(c)(1), (2) Date Sep. 30, 2020, PCT Pub. No. WO2019/195304, PCT Pub. Date Oct. 10, 2019.
Claims priority of provisional application 62/652,127, filed on Apr. 3, 2018.
Prior Publication US 2021/0145859 A1, May 20, 2021
Int. Cl. C07H 21/02 (2006.01); A61K 31/7105 (2006.01); A61K 47/46 (2006.01); A61P 21/00 (2006.01); A61P 25/00 (2006.01); C12N 15/113 (2010.01)

CPC A61K 31/7105 (2013.01) [A61K 47/46 (2013.01); A61P 21/00 (2018.01); A61P 25/00 (2018.01); C12N 15/113 (2013.01); C12N 2310/141 (2013.01)]

8 Claims

1. A method of delaying an onset of motor neuron (MN) degeneration, reducing a risk of developing MN degeneration, or treating amyotrophic lateral sclerosis (ALS) associated with an SOD1 gene mutation in an adult subject bearing the SOD1 gene mutation, comprising administering to a central nervous system of the adult subject a therapeutically or prophylactically effective amount of genes or transgenes containing members of mir-17˜92 cluster, wherein the members of the miR-17˜92 cluster are miR-17, miR-17⁺, miR-18a, miR-19a, miR-19b, miRr-20a, and miR-92a, and wherein the genes or transgenes are incorporated into or encapsulated in a viral vector.