| 发明名称 |
METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF NEUROLOGICAL PHENOTYPE ASSOCIATED WITH FRIEDREICH ATAXIA |
| 摘要 |
The present invention relates to a vector which comprises a nucleic acid sequence encoding for the frataxin (FXN) gene for use in the prevention and treatment of neurological phenotype associated with Friedreich ataxia in a subject in need thereof. |
| 申请公布号 |
WO2016150964(A1) |
申请公布日期 |
2016.09.29 |
| 申请号 |
WO2016EP56263 |
申请日期 |
2016.03.22 |
| 申请人 |
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE);CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS);UNIVERSITÉ DE STRASBOURG |
发明人 |
PUCCIO, Hélène;PIGUET, Françoise |
| 分类号 |
A61K48/00;C07K14/47;C12N15/86 |
主分类号 |
A61K48/00 |
| 代理机构 |
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