发明名称 |
GENE VECTOR |
摘要 |
PROBLEM TO BE SOLVED: To provide a novel gene vector for use in gene transfer and gene therapy.SOLUTION: A gene vector for use in gene therapy comprises 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.SELECTED DRAWING: Figure 1 |
申请公布号 |
JP2016028582(A) |
申请公布日期 |
2016.03.03 |
申请号 |
JP20150175986 |
申请日期 |
2015.09.07 |
申请人 |
OSPEDALE SAN RAFFAELE SRL;FONDAZIONE TELETHON |
发明人 |
ALESSANDRA BIFFI;BERNHARD RUDOLF GENTNER;LUIGI NALDINI |
分类号 |
C12N15/09;A61K31/7088;A61K35/12;A61K35/76;A61K48/00;A61P35/00;A61P37/00;A61P37/04;A61P43/00;C12N1/15;C12N1/19;C12N1/21;C12N5/10;C12N7/02;C12Q1/68 |
主分类号 |
C12N15/09 |
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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