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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Bibliografiske detaljer
Udgivet i:Mol Ther Nucleic Acids
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2016
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Original Article
Online adgang: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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