Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy.

Among the tested LV constructs, one miR-126 target (hereon referred to as hGALC_126T.LV) performed at best since it allowed a highly significant ( P < 0.0001) repression of hGALC activity in both CB- and BM-HSPCs at +1 day of culture ( Figure 1a ) and a sustained hGALC expression upon in vitro cell differentiation (after 14 days of liquid culture) ( Figure 1b ), in the presence of comparable vector copy number (VCN) values with the other tested constructs ( Supplementary Figure S1a ).

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