Fig. 5: SNP6-11A siRNA maintains selective-silencing of mutant HTT in neurons differentiated from human iPSCs. | Nature Communications

Fig. 5: SNP6-11A siRNA maintains selective-silencing of mutant HTT in neurons differentiated from human iPSCs.

From: Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models

Fig. 5

Western blot analysis of HD109 neurons, which are heterozygous at SNP2273, showed lowering of mutant HTT a compared to wild-type HTT b with SNP6-11. Cells were treated for 5 days with 3.0 µM siRNA. Pixel intensity of mutant or wild-type HTT was standardized to that of vinculin and reported as a percentage of untreated. Non-selective targeting with siRNAHTT was used as a positive control. A non-targeting control (NTC) siRNA was used as a negative control. One-way ANOVA and posthoc Tukey test; n = 10 biological replicates (wells) per group. Error bars extend to minimum and maximum value. The lower bound of the box is the 25th percentile and the upper is the 75th, with a line at the median. Source data are provided as a source data file.

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