I04 AAV5-MIHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in a huntington disease minipig model
Jiri Klima, Melvin M Evers, Jana Miniarikova, Stefan Juhas, Astrid Vallès, Bozena Bohuslavova, Jana Juhasova, Helena Kupcova Skalnikova, Petr Vodicka, Ivona Valekova, Cynthia Brouwers, Bas Blits, Jacek Lubelski, Hana Kovarova, Sander J van Deventer, Harald Petry, Jan Motlik, Pavlina Konstantinova, Zdenka Ellederova
Journal of Neurology, Neurosurgery & Psychiatry Sep 2018, 89 (Suppl 1) A89; DOI: 10.1136/jnnp-2018-EHDN.240