Helene Monique PUCCIO,Patrick AUBORG,Ronald G. CRYSTAL,Pierre BOUGNERES
申请号：
US15916907
公开号：
US20180334687A1
申请日:
2018.03.09
申请国别(地区):
US
年份:
2018
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.
