| 发明名称 |
METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO FRIEDREICH ATAXIA |
| 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). |
| 申请公布号 |
US2015313969(A1) |
申请公布日期 |
2015.11.05 |
| 申请号 |
US201514718696 |
申请日期 |
2015.05.21 |
| 申请人 |
Institut National de la Sante et de la Recherche Medicale (INSERM) ;CNRS (Centre National de la Recherche Scientifique) ;Universite de Strasbourg ;Cornell University ;Universite Paris-Sud XI ;Assistance Publique-Hopitaux de Paris (APHP) |
发明人 |
Puccio Helene Monique;Aubourg Patrick;Crystal Ronald G.;Bougneres Pierre |
| 分类号 |
A61K38/44;A61K9/00;C12N7/00 |
主分类号 |
A61K38/44 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method for preventing or treating cardiomyopathy due to energy failure in a subject in need thereof, comprising administering by intravenous injection to said subject a therapeutically effective amount of a vector which comprises a nucleic acid sequence of a gene that can reverse energy failure. |
| 地址 |
Paris FR |
