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. 2017 Jan 4;25(1):12–23. doi: 10.1016/j.ymthe.2016.11.010

Figure 5.

Figure 5

In Vivo Gene Editing of the Mutant HTT Allele

(A) Cartoon depicting rAAV shuttle vectors containing the SpCas9 and sgHD1/i3 expression cassettes (mCMV, minimal CMV promoter; mpA, minimal polyA and hU6p, human U6 promoter; pA, SV40 polyA). (B) PCR of isolated genomic DNA showing human HTT exon-1 targeted deletion after injection of vectors expressing SpCas9 and sgRNA sequences is shown. left striatum, LStr; right striatum, RStr. (C) qRT-PCR analysis of HTT mRNA levels in striatum samples harvested 3 weeks after SpCas9 and sgHD1/i3 delivery is shown. All of the samples were normalized to beta actin and the results are mean ± SEM relative to uninjected striatal samples (n = 7 animals per group, #p < 0.0001, and unpaired t test).