Nature Medicine11, 423 - 428 (2005)
Published online: 13 March 2005; | doi:10.1038/nm1207
Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS
Cédric Raoul1, Toufik Abbas-Terki1, Jean-Charles Bensadoun1, Sandrine Guillot1, Georg Haase2, Jolanta Szulc1, Christopher E Henderson3
& Patrick Aebischer1
1
Integrative Biosciences Institute, Ecole Polytechnique Fédérale de Lausanne (EPFL), CH-1015 Lausanne, Switzerland.
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 SOD1G93A 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.
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