Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model

@article{Ralph2005SilencingMS,
  title={Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model},
  author={Gareth S. Ralph and Pippa A. Radcliffe and Denise M Day and Janine M Carthy and Marie A Leroux and Debbie C. P. Lee and Liang-Fong Wong and Lynsey G. Bilsland and Linda Greensmith and Susan M. Kingsman and Kyriacos A. Mitrophanous and Nicholas D Mazarakis and Mimoun Azzouz},
  journal={Nature Medicine},
  year={2005},
  volume={11},
  pages={429-433}
}
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease resulting in the selective death of motor neurons in the brain and spinal cord. Some familial cases of ALS are caused by dominant mutations in the gene encoding superoxide dismutase (SOD1). The emergence of interfering RNA (RNAi) for specific gene silencing could be therapeutically beneficial for the treatment of such dominantly inherited diseases. We generated a lentiviral vector to mediate expression of RNAi molecules… CONTINUE READING

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