Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis

@article{Ding2003SelectiveSB,
  title={Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis},
  author={Hongliu Ding and Dianne S. Schwarz and Alex C. Keene and El Bachir Affar and L A Fenton and Xu-Gang Xia and Yang Shi and Phillip D. Zamore and Zuoshang Xu},
  journal={Aging Cell},
  year={2003},
  volume={2}
}
RNA interference (RNAi) can achieve sequence‐selective inactivation of gene expression in a wide variety of eukaryotes by introducing double‐stranded RNA corresponding to the target gene. Here we explore the potential of RNAi as a therapy for amyotrophic lateral sclerosis (ALS) caused by mutations in the Cu, Zn superoxide dismutase (SOD1) gene. Although the mutant SOD1 is toxic, the wild‐type SOD1 performs important functions. Therefore, the ideal therapeutic strategy should be to selectively… Expand
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