PMID: 16520821DOI: 10.1038/sj.gt.3302726Mar 8, 2006Paper
Target selectivity in mRNA silencing
Gene Therapy
N Aronin
Abstract
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.
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