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

EVERS, M., MINIARIKOVA, J., JUHÁS, Štefan, VALLÉS, A., BOHUSLAVOVÁ, Božena, JUHÁSOVÁ, Jana, KUPCOVÁ SKALNÍKOVÁ, Helena, VODIČKA, Petr, VALEKOVÁ, Ivona, BROUWERS, C., BLITS, B., LUBELSKI, J., KOVÁŘOVÁ, Hana, ELLEDEROVÁ, Zdeňka, VAN DEVENTER, S., PETRY, H., MOTLÍK, Jan, 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. Dostupné z: doi: 10.1016/j.ymthe.2018.06.021