Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing

Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing

Huntington’s disease (HD) is an autosomal dominant neurodegenerative disorder resulting from a polyglutamine expansion in the huntingtin (HTT) protein. There is currently no cure for this disease, but recent studies suggest that RNAi to downregulate the expression of both normal and mutant HTT is a...

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Bibliographic Details
Main Authors: Karine Cambon, Virginie Zimmer, Sylvain Martineau, Marie-Claude Gaillard, Margot Jarrige, Aurore Bugi, Jana Miniarikova, Maria Rey, Raymonde Hassig, Noelle Dufour, Gwenaelle Auregan, Philippe Hantraye, Anselme L. Perrier, Nicole Déglon
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Huntington
RNA interference
biosafety
off-targets
gene expression
iPSCs
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300669

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