| 发明名称 |
THERAPEUTIC STRATEGIES TO TREAT CNS PATHOLOGY IN MUCOPOLYSACCHARIDOSES |
| 摘要 |
The invention refers to nucleotide sequence encoding for a chimeric sulfatase, viral vectors expressing such sequences for gene therapy and pharmaceutical uses of the chimeric expressed protein. The invention is particularly applied in the therapy of mucopolysaccharidosis, preferably type IIIA. |
| 申请公布号 |
CA2822559(A1) |
申请公布日期 |
2012.06.28 |
| 申请号 |
CA20102822559 |
申请日期 |
2010.12.22 |
| 申请人 |
FONDAZIONE TELETHON |
发明人 |
BALLABIO, ANDREA;FRALDI, ALESSANDRO |
| 分类号 |
A61K48/00;A61K38/46;C12N15/62;C12N15/85;C12N15/861 |
主分类号 |
A61K48/00 |
| 代理机构 |
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