Ontology highlight
ABSTRACT:
SUBMITTER: Cambon K
PROVIDER: S-EPMC5453866 | biostudies-literature | 2017 Jun
REPOSITORIES: biostudies-literature
Cambon Karine K Zimmer Virginie V Martineau Sylvain S Gaillard Marie-Claude MC Jarrige Margot M Bugi Aurore A Miniarikova Jana J Rey Maria M Hassig Raymonde R Dufour Noelle N Auregan Gwenaelle G Hantraye Philippe P Perrier Anselme L AL Déglon Nicole N
Molecular therapy. Methods & clinical development 20170511
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 promising therapeutic approach. We previously developed a small hairpin RNA (shRNA), vectorized in an HIV-1-derived lentiviral vector (LV), that reduced pathology in an HD rodent model. Here, we modif ...[more]