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Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington s Disease Mice

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Academic year: 2021

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Figure

Figure 1  Mi2.4 demonstrates improved safety, relative to sh2.4, in  Hd-n171-82Q mice
Figure 3  Mi2.4 silences both mutant human and wild-type mouse  htt mrnAs in Hd-n171-82Q mice
Figure 4  Mi2.4 provides therapeutic benefit in Hd-n171-82Q mice. (a) Timeline for testing the therapeutic efficacy of AAV1-mi2.4 in HD-N171- HD-N171-82Q mice
Figure  5  transcriptional  changes  resulting  from  knockdown  of  wild-type  htt  in  mouse  striatum
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