Double-mismatched siRNAs enhance selective gene silencing of a mutant ALS-causing allele

  title={Double-mismatched siRNAs enhance selective gene silencing of a mutant ALS-causing allele},
  author={Chang-ming Geng and Hong-liu Ding},
  journal={Acta Pharmacologica Sinica},
AbstractAim:Our previous study demonstrated an siRNA-mediated, allele-specific silencing of mutant genes that cause amyotrophic lateral sclerosis. To improve siRNA design for better therapeutic use of RNA interference, we systematically tested the base-pairing mismatch strategy in the design of asymmetric siRNA.Methods:A naturally symmetric siRNA that targets the human Cu Zn superoxide dismutase G85R mutant allele was modified by placing either 1 or 2 mismatches at the end of the siRNA from… 
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  • V. Grinev
  • Biology, Medicine
    Molecular Biology
  • 2012
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