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. Author manuscript; available in PMC: 2011 Nov 30.
Published in final edited form as: Biochemistry. 2010 Nov 8;49(47):10166–10178. doi: 10.1021/bi101208k

Figure 1. Strategy for targeting the expanded CAG repeat region of mutant HTT mRNA to elicit allele-selective inhibition.

Figure 1

(A) Schematic of the HTT mRNA and the CAG repeat region that is targeted with complementary CTG-repeat ASOs. UTR, untranslated region. (B) Chemically modified base, backbone and nucleosides incorporated into ASOs tested for allele-selective inhibition of HTT expression.