L. V. D. Bent, C. Brouwers, Robin Pocornie, A. Stam, Steffi Jonk, J. Klíma, Š. Juhás, J. Motlík, M. Evers, Zdenka Ellederovaé, A. Vallès
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I04 Aav5-mihtt gene therapy mediates sustained mutant huntingtin lowering in brain and cerebrospinal fluid of Huntington disease minipigs up to 4 years