AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models.

Mol Ther Methods Clin Dev · 2019 · PMC6446047 · PMID 30984798

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a clear trendno p-value reported

A clear trend toward dose-dependent reduction of human mutant HTT was shown, with up to 85% lowering.

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