Skip to main content

Advertisement

Fig. 7 | Molecular Neurodegeneration

Fig. 7

From: Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

Fig. 7

Mutant huntingtin gene expression analysis after treatment with ZFs. a Linear regression showing negative correlations of mut HTT RNA levels and ZF-KOX1 expression 2, 4, and 6 weeks after treatment, suggesting an effective repression of mut HTT by the treatment. Black diamonds show the mean mut HTT expression values (±1 S.E.M.) of the control hemispheres of each group. ZF-KOX1 expression levels are in arbitrary units (a.u), normalised to the maximum ZF-KOX1 qRT-PCR signal across all samples. b Percentage of mut HTT with respect to the average value in the control hemispheres, over the same period. The data show an average of ~25 % reduction of mut HTT, 2 weeks post-treatment (previously reported in [14]), with an individual mouse showing up to ~40 % reduction. The average percentage increases with time, but later values should be interpreted cautiously because of ZF-KOX1 expression leakage to the contralateral hemisphere, and because of the significant neuronal loss. c Linear regression analysis testing for negative correlations between mut HTT RNA levels and mZF-KRAB expression, at 2, 4 and 6 weeks after treatment. mZF-KRAB expression levels are in arbitrary units (a.u), normalised to the maximum mZF-KRAB qRT-PCR signal across all samples. d Percentage of mut HTT with respect to the average value in the control hemispheres over the same period. The columns show mean RNA expression levels; error bars: ±1 S.E.M. *p < 0.05; § p < =0.06

Back to article page