Cornell University;INSERM (Institut National de la S;Centre National De La Recherche Scientifique (CNRS);Université Paris-Sud XI;APHP (Assistance Publique - Hôpitaux de Paris);Université de Strasbourg
发明人:
PUCCIO, Helene Monique,CRYSTAL, Ronald G.,BOUGNERES, Pierre,AUBOURG, Patrick
申请号：
AU2020200408
公开号：
AU2020200408A1
申请日:
2020.01.21
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
#$%^&*AU2020200408A120200213.pdf#####- '41 ABSTRACT OF THE INVENTION METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT 5 AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE The present invention relates 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 0 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.
