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
EP2424571(A2)
申请公布日期
2012.03.07
申请号
EP20100723305
申请日期
2010.04.30
申请人
FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR;FONDAZIONE TELETHON
发明人
BIFFI, ALESSANDRA;GENTNER, BERNHARD, RUDOLF;NALDINI, LUIGI
