- 专利标题： METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
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申请号： EP23201456.3申请日： 2014-01-31
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公开(公告)号： EP4324845A3公开(公告)日： 2024-04-17
- 发明人: PUCCIO, Hélène Monique , AUBOURG, Patrick , CRYSTAL, Ronald G , BOUGNERES, Pierre
- 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-Saclay , APHP (Assistance Publique - Hôpitaux de Paris)
- 申请人地址： FR 75013 Paris 101 rue de Tolbiac
- 专利权人： 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-Saclay,APHP (Assistance Publique - Hôpitaux de Paris)
- 当前专利权人： 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-Saclay,APHP (Assistance Publique - Hôpitaux de Paris)
- 当前专利权人地址： FR 75013 Paris 101 rue de Tolbiac
- 代理机构： Cooley (UK) LLP
- 优先权： US 201313756651 2013.02.01
- 分案原申请号： 14702565.4 2014.01.31
- 主分类号： A61K31/7088
- IPC分类号： A61K31/7088 ; A61K38/44 ; C07K14/47 ; A61K48/00 ; A61P9/00
摘要：
The present invention relates 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.
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.
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