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

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    Evers, M., Miniarikova, J., Juhás, Š., Vallés, A., Bohuslavová, B., Juhásová, J., Kupcová Skalníková, H., Vodička, P., Valeková, I., Brouwers, C., Blits, B., Lubelski, J., Kovářová, H., Ellederová, Z., van Deventer, S., Petry, H., Motlík, J., Konstantinová, P. AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington's Disease Minipig Model. Molecular Therapy. 2018, 26(9), 2163-2177. ISSN 1525-0016. E-ISSN 1525-0024. Available: https://doi.org/10.1016/j.ymthe.2018.06.021
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