Lentiviral-mediated silencing of SOD1-through RNA interference retards disease onset and progression in a mouse model of ALS

• Autores: Cédric Raoul, Toufik Abbas-Terki, Jean-Charles Bensadoun, Christopher E. Henderson, Patrick Aebischer
• Localización: Nature medicine, ISSN 1078-8956, Nº. 4, 2005, págs. 423-428
• Idioma: inglés
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• Resumen

  • 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.