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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Bibliografiset tiedot
Julkaisussa:Mol Ther Methods Clin Dev
Päätekijät: 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
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: American Society of Gene & Cell Therapy 2017
Aiheet:
Original Article
Linkit: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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