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
Publicado en:Mol Ther Nucleic Acids
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: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2016
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC5014463/
https://ncbi.nlm.nih.gov/pubmed/27003755
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.7
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