AAV5-MIHTT GENE THERAPY DEMONSTRATES BROAD DISTRIBUTION AND STRONG HUMAN MUTANT HUNTINGTIN LOWERING IN A HUNTINGTON DISEASE MINIPIG MODEL

Klíma, J., Evers, M., Miniariková, J., Juhás, Š., Vallés, A., Bohuslavová, B., Juhásová, J., Kupcová Skalníková, H., Vodička, P., Valeková, I., Brouwers, C., Blits, B., Lubelski, J., Kovářová, H., van Deventer, S., Petry, H., Motlík, J., Konstantinová, P., Ellederová, Z. AAV5-MIHTT GENE THERAPY DEMONSTRATES BROAD DISTRIBUTION AND STRONG HUMAN MUTANT HUNTINGTIN LOWERING IN A HUNTINGTON DISEASE MINIPIG MODEL. Journal of Neurology Neurosurgery and Psychiatry. 2018, 89(S1), A89-A89. ISSN 0022-3050. E-ISSN 1468-330X. Dostupné z: doi: 10.1136/jnnp-2018-EHDN.240