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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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Cambon, Karine, Zimmer, Virginie, Martineau, Sylvain, Gaillard, Marie-Claude, Jarrige, Margot, Bugi, Aurore, Miniarikova, Jana, Rey, Maria, Hassig, Raymonde, Dufour, Noelle, Auregan, Gwenaelle, Hantraye, Philippe, Perrier, Anselme L., Déglon, Nicole
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5453866/
https://ncbi.nlm.nih.gov/pubmed/28603746
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.05.001
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