Article
In vivo application of an RNAi strategy for the selective suppression of a mutant allele.
Human gene therapy - 1 Jan 2011
Kubodera Takayuki, Yamada Hiromi, Anzai Masayuki, Ohira Shinga, Yokota Shigefumi, Hirai Yukihiko, Mochizuki Hideki, Shimada Takashi, Mitani Tasuku, Mizusawa Hidehiro, Yokota Takanori
Abstract excerpt
Gene therapy for dominantly inherited diseases with small interfering RNA (siRNA) requires mutant allele-specific suppression when genes in which mutation causes disease normally have an important role. We previously proposed a strategy for selective suppression of mutant alleles; both mutant and wild-type alleles are inhibited by most effective siRNA, and wild-type protein is restored using mRNA mutated to be...
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