Raoul, C.Abbas-Terki, T.Bensadoun, J.C.Guillot, S.Haase, G.Szulc, J.Henderson, C. E.Aebischer, P.2007-03-092007-03-092007-03-09200510.1038/nm1207https://infoscience.epfl.ch/handle/20.500.14299/3779WOS:0002281805000346563Mutations 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.Amyotrophic Lateral Sclerosis/ geneticsAnimalsDisease ModelsAnimalDisease ProgressionGenetic VectorsHumansLentivirusMiceMiceTransgenicMolecular Sequence DataMutationRNA InterferenceRNASmall InterferingSuperoxide Dismutase/ geneticsAnimalMicetext::journal::journal article::research article