Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression
Author: ["Cédric Raoul","Toufik Abbas-Terki","Jean-Charles Bensadoun","Sandrine Guillot","Georg Haase","Jolanta Szulc","Christopher E Henderson","Patrick Aebischer"]
Publication: Nature Medicine
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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 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.
Cite this article
Raoul, C., Abbas-Terki, T., Bensadoun, JC. et al. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat Med 11, 423–428 (2005). https://doi.org/10.1038/nm1207
