Target selectivity in mRNA silencing.
Despite the excitement and promise of RNA interference in treating neurodegenerative disease, disease gene mRNA might resist mRNA silencing. Conventional siRNA design does not uniformly distinguish a mutant from a wild-type allele. CAG expansions in trinucleotide repeat diseases are unselective targets for small siRNAs. This review will consider recent discoveries in mechanisms of RNA interference and siRNA modifications that improve siRNA selectivity, delivery and performance.
Aronin
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University of Massachusetts, Worcester, MA 1655, USA. Aronin@umassmed.edu
D000818:Animals; D018390:Gene Targeting / Q000379:methods; D005818:Genetic Engineering; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D006816:Huntington Disease / Q000628:therapy; D009154:Mutation; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000235:genetics*; D034743:RNA-Induced Silencing Complex / Q000235:genetics
D034741:RNA, Small Interfering; D034743:RNA-Induced Silencing Complex