| 发明名称 |
GENE VECTOR |
| 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 |
EA023938(B1) |
申请公布日期 |
2016.07.29 |
| 申请号 |
EA20110071335 |
申请日期 |
2010.04.30 |
| 申请人 |
OSPEDALE SAN RAFFAELE S.R.L.;FONDAZIONE TELETHON |
发明人 |
BIFFI ALESSANDRA;GENTNER BERNHARD, RUDOLF;NALDINI LUIGI |
| 分类号 |
A61K48/00;A61P7/00;A61P35/00;C12N15/113;C12N15/63;C12N15/85 |
主分类号 |
A61K48/00 |
| 代理机构 |
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