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.
申请公布号
SG10201610827R(A)
申请公布日期
2017.02.27
申请号
SG10201610827R
申请日期
2010.04.30
申请人
OSPEDALE SAN RAFFAELE S.R.L;FONDAZIONE TELETHON
发明人
BIFFI, ALESSANDRA;GENTNER, BERNHARD RUDOLF;NALDINI, LUIGI