Helene Monique Puccio,Patrick Aubourg,Ronald G. Crystal,Pierre Bougneres
申请号：
US13756651
公开号：
US09066966B2
申请日:
2013.02.01
申请国别(地区):
US
年份:
2015
代理人:
摘要：
A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof is provided. The method comprises administering to the subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence encoding a gene that can reverse energy failure. An exemplary cardiomyopathy is that which is associated with Friedreich ataxia and an exemplary nucleic acid sequence comprises a nucleic acid that encodes frataxin (FXN).
