US 12,138,298 B2
Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
Sandro Alves, Paris (FR); Nathalie Cartier-Lacave, Paris (FR); Luis Pereira De Almeida, Coimbra (PT); Clévio Nobrega, Coimbra (PT); and Liliana Mendonça, Coimbra (PT)
Assigned to BRAINVECTIS, Paris (FR); INSERM (INSTITUT NATIONAL DE LA SANTËET DE LA RECHERCHE MÊDICALE), Paris (FR); UNIVERSIDADE DE COIMBRA, Coimbra (PT); and CENTRO DE NEUROCIÊNCIAS BIOLOGIA CELULAR, Coimbra (PT)
Appl. No. 16/480,541
Filed by BRAINVECTIS, Paris (FR); INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE), Paris (FR); UNIVERSIDADE DE COIMBRA, Coimbra (PT); and CENTRO DE NEUROCIÊNCIAS E BIOLOGIA CELULAR, Coimbra (PT)
PCT Filed Jan. 30, 2018, PCT No. PCT/EP2018/052199
§ 371(c)(1), (2) Date Jul. 24, 2019,
PCT Pub. No. WO2018/138371, PCT Pub. Date Aug. 2, 2018.
Claims priority of application No. 17305100 (EP), filed on Jan. 30, 2017.
Prior Publication US 2019/0343927 A1, Nov. 14, 2019
Int. Cl. A61K 38/44 (2006.01); A61K 48/00 (2006.01); A61P 25/28 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01)
CPC A61K 38/44 (2013.01) [A61K 48/0075 (2013.01); A61P 25/28 (2018.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); C12Y 114/13098 (2013.01); A61K 48/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01)] 14 Claims
 
1. A method of treatment of a Polyglutamine repeat spinocerebellar ataxia, comprising administering a vector which comprises cholesterol 24-hydroxylase encoding nucleic acid to a patient in need thereof.