AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease

Huntington’s disease (HD) is a fatal progressive neurodegenerative disorder caused by a mutation in the huntingtin (HTT) gene. To date, there is no treatment to halt or reverse the course of HD. Lowering of either total or only the mutant HTT expression is expected to have therapeutic benefit. This...

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Опубликовано в: :Gene Ther
Главные авторы: Miniarikova, J, Zimmer, V, Martier, R, Brouwers, C C, Pythoud, C, Richetin, K, Rey, M, Lubelski, J, Evers, M M, van Deventer, S J, Petry, H, Déglon, N, Konstantinova, P
Формат: Artigo
Язык:Inglês
Опубликовано: Nature Publishing Group 2017
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Original Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5658675/
https://ncbi.nlm.nih.gov/pubmed/28771234
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2017.71
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