Combination of stem cell and gene therapy ameliorates symptoms in Huntington’s disease mice

Suppression of HTT by expressing shHD. a qRT-PCR analysis of HTT exon 1 expression of HD-shHD-NPC showing significant suppression of HTT in HD-shHD-NPC compared to both HD-NPC (P？<？0.0001) and WT-NPC (P？=？0.003). Results shown from three biological replicates. Data is represented as mean？±？SEM (*P？<？0.05, ***P？<？0.0001; ANOVA). b Western blot analysis showing increased oligomeric mHTT aggregates in stacking gel (mEM48) and soluble form (1C2) in HD-NPC while expression of shHD in HD-NPC decreased both aggregate accumulation in the stacking gel and soluble form of mHTT. Positive control used in this study was 293FT cells overexpressing expanded poly-Q (84Q) under the control of ubiquitin promote