US 12,121,563 B2
Rescuing voltage-gated sodium channel function in inhibitory neurons
John K. Mich, Seattle, WA (US); Edward Sebastian Lein, Mercer Island, WA (US); Jonathan Ting, Lake Forest Park, WA (US); Boaz P. Levi, Seattle, WA (US); Erik Hess, Issaquah, WA (US); and Franck Kalume, Bothell, WA (US)
Assigned to Allen Institute, Seattle, WA (US); and Seattle Children's Hospital, Seattle, WA (US)
Appl. No. 17/044,232
Filed by ALLEN INSTITUTE, Seattle, WA (US); and SEATTLE CHILDREN'S HOSPITAL, Seattle, WA (US)
PCT Filed Apr. 9, 2019, PCT No. PCT/US2019/026638
§ 371(c)(1), (2) Date Sep. 30, 2020,
PCT Pub. No. WO2019/199867, PCT Pub. Date Oct. 17, 2019.
Claims priority of provisional application 62/810,281, filed on Feb. 25, 2019.
Claims priority of provisional application 62/742,835, filed on Oct. 8, 2018.
Claims priority of provisional application 62/655,043, filed on Apr. 9, 2018.
Prior Publication US 2021/0015898 A1, Jan. 21, 2021
Int. Cl. A61K 38/17 (2006.01); A61K 9/00 (2006.01); A61K 48/00 (2006.01); C07K 14/705 (2006.01); C12N 15/86 (2006.01)
CPC A61K 38/177 (2013.01) [A61K 9/0019 (2013.01); A61K 48/0058 (2013.01); C07K 14/705 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01)] 20 Claims
 
1. An expression construct comprising (i) an enhancer having the sequence as set forth in SEQ ID NO: 3; (ii) a promoter having the sequence as set forth in SEQ ID NO: 30 or SEQ ID NO: 31; and (iii) a coding sequence encoding a protein that rescues voltage-gated sodium channel function.