BIFFI, Alessandra,GENTNER, Bernhard,NALDINI, Luigi
申请号：
LT10723305
公开号：
LT2424571T
申请日:
2010.04.30
申请国别(地区):
LT
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.
