Figure 1.

In Vitro Reduction of PTB Protein by siRNAs and miRNAs and Long-Term Selectivity of the Olig001 AAV Vector in the Rat Striatum

(A) The left western blot shows that siRNAs 3 and 4 completely prevented PTB protein expression following transfection of HeLa cells with the siRNA and a rat ptbp expression plasmid. The right western blot shows that conversion of siRNAs 3 and 4 into miRNAs 3 and 4, followed by packaging into recombinant AAV2 virus, prevents PTB protein expression in HEK293 cells. (B) The oligodendrocyte preference for the Olig001 AAV-GFP vector in the rat striatum 10 days post-transduction is shown. GFP-positive cells co-localized with Olig2-positive cells, but they did not co-localize with NeuN- or GFAP-positive cells. (C) The same transduction pattern from the Olig001 AAV-GFP vector 6 months after striatal transduction in the rat also is shown.