Fig. 4: SNP6-11 siRNA maintains selective-silencing of mutant HTT in patient-derived NSCs. | Nature Communications

Fig. 4: SNP6-11 siRNA maintains selective-silencing of mutant HTT in patient-derived NSCs.

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

Fig. 4

HD patient derived neural stem cells carrying an A or a G at SNP site rs362273 (a) were treated for 5 days with SNP6-11 or non-selective siRNAHTT at the concentrations listed. Graphs show pixel intensity quantification of mutant (b), or wild-type (c) HTT signal intensity standardized to that of vinculin reported as a percentage of untreated NSCs. A non-targeting control (NTC) siRNA was used as a negative control. Non-selective targeting with siRNAHTT was used as a positive control. One-way ANOVA and posthoc Tukey test; n = 5 wells for siRNAHTT at 3.0 and 4.0 µM, n = 8 wells per group for the remaining treatments. 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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