title: Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS.
abstract: Mutations in Cu/Zn superoxide dismutase (encoded by SOD1), one of the causes of familial amyotrophic lateral sclerosis (ALS), lead to progressive death of motoneurons through a gain-of-function mechanism. RNA interference (RNAi) mediated by viral vectors allows for long-term reduction in gene expression and represents an attractive therapeutic approach for genetic diseases characterized by acquired toxic properties. We report that in SOD1(G93A) transgenic mice, a model for familial ALS, intraspinal injection of a lentiviral vector that produces RNAi-mediated silencing of SOD1 substantially retards both the onset and the progression rate of the disease.
authors.affiliation: Integrative Biosciences Institute, Ecole Polytechnique Fédérale de Lausanne, Switzerland.
authors.forename: Cédric
authors.initials: C
authors.lastname: Raoul
authors.forename: Toufik
authors.initials: T
authors.lastname: Abbas-Terki
authors.forename: Jean-Charles
authors.initials: JC
authors.lastname: Bensadoun
authors.forename: Sandrine
authors.initials: S
authors.lastname: Guillot
authors.forename: Georg
authors.initials: G
authors.lastname: Haase
authors.forename: Jolanta
authors.initials: J
authors.lastname: Szulc
authors.forename: Christopher E
authors.initials: CE
authors.lastname: Henderson
authors.forename: Patrick
authors.initials: P
authors.lastname: Aebischer
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000235:genetics*; D000818:Animals; D004195:Disease Models, Animal; D018450:Disease Progression; D005822:Genetic Vectors; D006801:Humans; D016086:Lentivirus; D051379:Mice; D008822:Mice, Transgenic; D008969:Molecular Sequence Data; D009154:Mutation; D034622:RNA Interference; D034741:RNA, Small Interfering; D013482:Superoxide Dismutase / Q000235:genetics*
chemical_list: D034741:RNA, Small Interfering; C109415:SOD1 G93A protein; D013482:Superoxide Dismutase