Ontology highlight
ABSTRACT:
SUBMITTER: Evers MM
PROVIDER: S-EPMC6127509 | biostudies-literature | 2018 Sep
REPOSITORIES: biostudies-literature
Evers Melvin M MM Miniarikova Jana J Juhas Stefan S Vallès Astrid A Bohuslavova Bozena B Juhasova Jana J Skalnikova Helena Kupcova HK Vodicka Petr P Valekova Ivona I Brouwers Cynthia C Blits Bas B Lubelski Jacek J Kovarova Hana H Ellederova Zdenka Z van Deventer Sander J SJ Petry Harald H Motlik Jan J Konstantinova Pavlina P
Molecular therapy : the journal of the American Society of Gene Therapy 20180625 9
Huntington's disease (HD) is a fatal neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the huntingtin gene. Previously, we showed strong huntingtin reduction and prevention of neuronal dysfunction in HD rodents using an engineered microRNA targeting human huntingtin, delivered via adeno-associated virus (AAV) serotype 5 vector with a transgene encoding an engineered miRNA against HTT mRNA (AAV5-miHTT). One of the challenges of rodents as a model of neurodegenerative di ...[more]