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
Published in: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
Language:Inglês
Published: American Society of Gene & Cell Therapy 2017
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Original Article
Online Access: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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