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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Methods Clin Dev
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2017
Pynciau:
Original Article
Mynediad Ar-lein: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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