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

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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Bibliographic Details
Main Authors: Jana Miniarikova, Ilaria Zanella, Angelina Huseinovic, Tom van der Zon, Evelyn Hanemaaijer, Raygene Martier, Annemart Koornneef, Amber L Southwell, Michael R Hayden, Sander J van Deventer, Harald Petry, Pavlina Konstantinova
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
adeno-associated virus
gene silencing
humanized HD mouse model
Huntington's disease
therapeutic microRNAs
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117300987

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