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 代理人
主权项
地址