Article
Therapy for dominant inherited diseases by allele-specific RNA interference: successes and pitfalls.
Current gene therapy - 1 Jan 2015
Trochet Delphine, Prudhon Bernard, Vassilopoulos Stéphane, Bitoun Marc
Abstract excerpt
RNA interference (RNAi) is a conserved mechanism for post-transcriptional gene silencing mediated by messenger RNA (mRNA) degradation. RNAi is commonly induced by synthetic siRNA or shRNA which recognizes the targeted mRNA by base pairing and leads to target-mRNA degradation. RNAi may discriminat...
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