AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model.

Mol Ther · 2018 · PMC6127509 · PMID 30007561

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a clear trendno p-value reported

Although the sample size in this study was limited, we observed a clear trend toward a dose-dependent reduction of human mutant huntingtin after AAV5-miHTT administration.

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