公开(公告)号：US20200370069A1

公开(公告)日：2020-11-26

申请号：US16629339

申请日：2018-07-06

Applicant: GENETHON ,  INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) ,  UNIVERSITE D'EVRY VAL D'ESSONNE

Inventor： ANA BUJ BELLO ,  MARTINA MARINELLO ,  SAMIA MARTIN

IPC: C12N15/86 ,  A61P25/00 ,  C07K14/47 ,  A61K38/17 ,  A61K48/00

Abstract: The present invention relates to a recombinant adeno-associated virus (rAAV) vector comprising a serotype 9 or rh10 AAV capsid, for use in a method for the treatment of spinal muscular atrophy (SMA).

公开(公告)号：US20230032826A1

公开(公告)日：2023-02-02

申请号：US17785422

申请日：2020-12-18

Applicant: GENETHON

Inventor： SERGE BRAUN ,  FLORIAN CARDON ,  YOHANN DICKX ,  MARINA GUILLET ,  SAMIA MARTIN ,  MARIE RANDOUX

IPC: C12N15/82 ,  C12N15/86

Abstract: The present invention relates to a method for producing a recombinant viral vector from hairy roots of a plant, in particular from hairy roots of a plant belonging to the Brassicaceae family. The invention also relates to a transgenic plant, a hairy root culture and a recombinant viral vector obtainable by the method of the invention.