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

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, deliv...

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Detalles Bibliográficos
Autores principales: Evers, Melvin M., Miniarikova, Jana, Juhas, Stefan, Vallès, Astrid, Bohuslavova, Bozena, Juhasova, Jana, Skalnikova, Helena Kupcova, Vodicka, Petr, Valekova, Ivona, Brouwers, Cynthia, Blits, Bas, Lubelski, Jacek, Kovarova, Hana, Ellederova, Zdenka, van Deventer, Sander J., Petry, Harald, Motlik, Jan, Konstantinova, Pavlina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6127509/
https://www.ncbi.nlm.nih.gov/pubmed/30007561
http://dx.doi.org/10.1016/j.ymthe.2018.06.021

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