Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS
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.
Keywords: Amyotrophic Lateral Sclerosis/ genetics ; Animals ; Disease Models ; Animal ; Disease Progression ; Genetic Vectors ; Humans ; Lentivirus ; Mice ; Mice ; Transgenic ; Molecular Sequence Data ; Mutation ; RNA Interference ; RNA ; Small Interfering ; Superoxide Dismutase/ genetics ; Animal ; Mice
Integrative Biosciences Institute, Ecole Polytechnique Federale de Lausanne, Switzerland.
Record created on 2007-03-09, modified on 2016-08-08