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. 2015 Dec 1;4(12):e266. doi: 10.1038/mtna.2015.38

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Copyright © 2015 Official journal of the American Society of Gene & Cell Therapy

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HTT10150 effectively silences huntingtin mRNA ipsilateral to the site of injection. HTT10150 was unilaterally injected into the striatum of WT (FVB/NJ) mice (2 µl). Mice were sacrificed at 5 days. Brains were sliced into 300-μm sections and six 2-mm punch biopsies of the (a) striatum and (b) cortex were collected from both ipsilateral and contralateral sides. Level of huntingtin mRNA was measured using QuantiGene (Affymetrix) normalized to housekeeping gene, Ppib (cyclophillin B), and presented as percent of untreated control (n = 8 mice, mean ± SD, three biopsies per region). P values are all calculated for each dose group relative to NTC by Two-way repeated-measures analysis of variance: 25 µg striatum, P < 0.0001; 12.5 µg striatum, P < 0.0001, P = 0.0002; 6.3 µg cortex, P = 0.0009. NTC, nontargeting control.