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Fig. 8 | Molecular Neurodegeneration

Fig. 8

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

Fig. 8

Long-term effects of bilateral intraventricular injection of AAV expressing mZF-KRAB under pCAG or pNSE promoters. a Zinc finger expression over time. mZF-KRAB transcript levels from whole brains were assayed by qRT-PCR at 3, 6, 12 and 24 weeks after viral (or PBS control) injections, in WT or R6/1 neonates. b Zinc finger repression of mutant Huntingtin in R6/1 mice. mut HTT (exon 1) expression levels in the whole brain samples from the various treatments were compared to transcript levels in PBS controls, by qRT-PCR. c Verification of lack of cross-reactivity of mZF-KRAB with short WT Htt alleles. WT Htt (exon 1) expression levels were quantified in the same treatment samples as above. Housekeeping genes and other control data are shown in Additional file 5. Error bars are S.E.M (n = 3). ** p < 0.01, *** p < 0.001, n.s. = not significant

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Molecular Neurodegeneration

ISSN: 1750-1326