Biffi, Alessandra,Gentner, Bernhard Rudolf,Naldini, Luigi
申请号：
HRP20200647
公开号：
HRP20200647T1
申请日:
2020.04.22
申请国别(地区):
HR
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.
