FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR;FONDAZIONE TELETHON
发明人:
申请号：
IL21580411
公开号：
IL215804D0
申请日:
2011.10.23
申请国别(地区):
IL
年份:
2012
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.
