UNIVERSITE PARIS-SUD XI;INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE);CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS);UNIVERSITE DE STRASBOURG;CORNELL UNIVERSITY;APHP (ASSISTA
发明人:
PUCCIO, HELENE MONIQUE,AUBOURG, PATRICK,CRYSTAL, RONALD G.,BOUGNERES, PIERRE
申请号：
CA2900135
公开号：
CA2900135A1
申请日:
2014.01.31
申请国别(地区):
CA
年份:
2014
代理人:
摘要：
The present invention relates to a method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can restore energy failure. More particularly, the invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.
