Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease

Huntington's disease (HD) is a neurodegenerative disorder caused by accumulation of CAG expansions in the huntingtin (HTT) gene. Hence, decreasing the expression of mutated HTT (mtHTT) is the most upstream approach for treatment of HD. We have developed HTT gene-silencing approaches based on ex...

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Detalles Bibliográficos
Autores principales: Miniarikova, Jana, Zanella, Ilaria, Huseinovic, Angelina, van der Zon, Tom, Hanemaaijer, Evelyn, Martier, Raygene, Koornneef, Annemart, Southwell, Amber L, Hayden, Michael R, van Deventer, Sander J, Petry, Harald, Konstantinova, Pavlina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5014463/
https://www.ncbi.nlm.nih.gov/pubmed/27003755
http://dx.doi.org/10.1038/mtna.2016.7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5014463/
https://www.ncbi.nlm.nih.gov/pubmed/27003755
http://dx.doi.org/10.1038/mtna.2016.7

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