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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
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Original Article
Acesso em linha: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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