BIFFI, Alessandra,GENTNER, Bernhard, Rudolf,NALDINI, Luigi
申请号：
DK10723305
公开号：
DK2424571T3
申请日:
2010.04.30
申请国别(地区):
DK
年份:
2020
代理人:
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.
