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. 2017 Sep 14;24(10):630–639. doi: 10.1038/gt.2017.71

Figure 3.

Figure 3

HTT mRNA knock-down induced by AAV5-miHTT-451 and AAV5-miSNP50T-451 vectors in the striatum of HD rats. (a) The structure and sequence of the hsa-pre-miR-451a precursor used in this study with the miRBase-predicted guide strand highlighted in pink (www.mirbase.org). (b) Schematic representation of the AAV5-miHTT-451 and AAV5-miSNP50T-451 expression cassettes; and LV-mtHTT-50C and LV-mtHTT-50T encoding a chimeric mutant HTT sequence with either C or T isoform of SNP rs362331. (c) Bilateral co-injections in the striatum (STR) of rats with LV-mtHTT-50C or LV-mtHTT-50T, and AAV5-miHTT-451 or AAV5-miSNP50T-451 vectors. The experimental groups and injection sites are outlined. (d) qPCR to determine AAV5 genome copies (gc) in the striatum of AAV5-miHTT-451 and AAV5-miSNP50T-451 injected rats (n=3), two months post-injection. Primers directed to the CAG promoter were used and the gc values were calculated based on the standard curve and considering the background signal from the negative control. (e) TaqMan qPCR assay shows HTT mRNA knock-down in the striatum (n=2-3) induced by the AAV5-miHTT-451 and AAV5-miSNP50T-451 expression products. Human HTT-specific exon-spanning primers were used and HTT values were subsequently normalized to GAPDH, an internal control set at 100%. All data were analyzed using one-way ANOVA. *P⩽0.05; **P⩽0.01; ***P⩽0.001; ****P⩽0.0001. The values were calculated as a mean±s.d.