An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment.

Time point analysis at 14 weeks of age showed that body weight ( Figure 4 E), grip strength ( Figure 4 F), and rotarod performance ( Figure 4 G) of S9-SNA-treated AR97Q mice were improved toward wild-type levels, although the improvements in body weight and rotarod performance did not reach statistical significance.

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