Article
Double-mismatched siRNAs enhance selective gene silencing of a mutant ALS-causing allele.
Acta pharmacologica Sinica - 1 Feb 2008
Geng Chang-ming, Ding Hong-liu
Abstract excerpt
AIM: 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...
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