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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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5658675/
https://www.ncbi.nlm.nih.gov/pubmed/28771234
http://dx.doi.org/10.1038/gt.2017.71

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5658675/
https://www.ncbi.nlm.nih.gov/pubmed/28771234
http://dx.doi.org/10.1038/gt.2017.71

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