US 12,331,101 B2
Treatment of spinal and bulbar muscular atrophy
Carlo Rinaldi, Oxford (GB); Wooi Fang Lim, Oxford (GB); and Matthew Wood, Oxford (GB)
Assigned to OXFORD UNIVERSITY INNOVATION LIMITED, Oxford (GB)
Appl. No. 17/765,116
Filed by Oxford University Innovation Limited, Oxford (GB)
PCT Filed Oct. 2, 2020, PCT No. PCT/GB2020/052435
§ 371(c)(1), (2) Date Mar. 30, 2022,
PCT Pub. No. WO2021/064421, PCT Pub. Date Apr. 8, 2021.
Claims priority of application No. 1914296.7 (GB), filed on Oct. 3, 2019.
Prior Publication US 2022/0380438 A1, Dec. 1, 2022
Int. Cl. C12N 15/11 (2006.01); A61K 48/00 (2006.01); A61P 21/00 (2006.01); C07K 14/075 (2006.01); C07K 14/72 (2006.01); C12N 15/86 (2006.01)
CPC C07K 14/721 (2013.01) [A61K 48/0058 (2013.01); A61P 21/00 (2018.01); C07K 14/075 (2013.01); C12N 15/86 (2013.01)] 5 Claims
 
1. A method for treatment of spinal and bulbar muscular atrophy (SBMA) in a patient, comprising administering to the patient a therapeutically effective amount of an expression construct encoding androgen receptor splice variant 2 (AR2), wherein the patient is a human patient having expansion of the polyglutamine tract in the N terminus domain (NTD) of androgen receptor (AR), wherein the expansion causes SBMA.