FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR;FONDAZIONE TELETHON
发明人:
ALESSANDRA BIFFI,BERNHARD RUDOLF GENTNER,LUIGI NALDINI
申请号：
BRPI1010873
公开号：
BRPI1010873A2
申请日:
2010.04.30
申请国别(地区):
BR
年份:
2016
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.
