RNA interference: From gene silencing to gene-specific therapeutics

摘要: In the past 4 years, RNA interference (RNAi) has become widely used as an experimental tool to analyse function of mammalian genes, both in vitro and vivo. By harnessing evolutionary conserved endogenous biological pathway, first identified plants lower organisms, double-stranded (dsRNA) reagents are bind promote degradation target RNAs, resulting knockdown expression specific genes. RNAi can be induced cells by introduction synthetic small interfering RNAs (siRNAs) 21–23 base pairs (bp) length or plasmid viral vector systems that express short hairpin (shRNAs) subsequently processed siRNAs cellular machinery. been define functional roles individual particularly disease. addition, siRNA shRNA libraries have developed allow systematic analysis genes required for disease processes such cancer using high throughput screens. gene animals, with development tissue-specific inducible promising provide a quicker cheaper way generate transgenic animals than conventional approaches. Finally, because ability silence disease-associated tissue culture animal models, RNAi-based clinical applications is gathering pace, technological enhancements improve stability delivery vivo, while minimising off-target nonspecific effects, developed.