Cornell University;INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (INSERM);CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS);UNIVERSITÉ PARIS-SUD XI;Assistance Pu;UNIVERSITÉ DE STRASBOURG
发明人:
Hélène Monique PUCCIO,Patrick AUBORG,Ronald G. CRYSTAL,Pierre BOUGNERES
申请号：
US16411997
公开号：
US20200157564A1
申请日:
2019.05.14
申请国别(地区):
US
年份:
2020
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).
