发明申请
US20200157564A1 METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
审中-公开
基本信息:
- 专利标题： METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
- 申请号：US16411997 申请日：2019-05-14
- 公开(公告)号：US20200157564A1 公开(公告)日：2020-05-21
- 发明人： Hélène Monique PUCCIO , Patrick AUBORG , Ronald G. CRYSTAL , Pierre BOUGNERES
- 申请人： INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE (INSERM) , CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) , UNIVERSITÉ DE STRASBOURG , Cornell University , UNIVERSITÉ PARIS-SUD XI , Assistance Publique-Hopitaux de Paris (APHP)
- 主分类号： C12N15/86
- IPC分类号： C12N15/86 ; A61K31/7088 ; A61K38/44 ; C12N7/00 ; A61K9/00 ; A61K48/00 ; C07K14/47
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).