Cornell University;Centre National de;Inserm (Institut National de la Sante et de la Recherche Medicale);Universite de Strasbourg;Universite Paris-Sud Xl;APHP (Assistance publique – Hopitaux de Paris)
发明人:
Puccio, Helene Monique,Aubourg, Patrick,Crystal, Ronald G.,Bougneres, Pierre
申请号：
AU2014211315
公开号：
AU2014211315A1
申请日:
2014.01.31
申请国别(地区):
AU
年份:
2015
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.
