| 发明名称 |
vetor genético |
| 摘要 |
A gene vector for use in gene therapy comprising at least one miRNA sequence target operably linked to a nucleotide sequence having a corresponding miRNA in a hematopoietic progenitor cell (HSPC) or hematopoietic stem cell (HSC) which prevents or reduces expression of the nucleotide sequence in a HSPC or HSC but not in a differentiated cell. |
| 申请公布号 |
BRPI1010873(A2) |
申请公布日期 |
2016.03.15 |
| 申请号 |
BR2010PI10873 |
申请日期 |
2010.04.30 |
| 申请人 |
FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR;FONDAZIONE TELETHON |
发明人 |
ALESSANDRA BIFFI;BERNHARD RUDOLF GENTNER;LUIGI NALDINI |
| 分类号 |
C12N15/63;A61K48/00;A61P7/00;A61P35/00;C12N15/113;C12N15/85 |
主分类号 |
C12N15/63 |
| 代理机构 |
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代理人 |
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| 主权项 |
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| 地址 |
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