Unlocking the potential of RNA interference as a therapeutic tool

Malta medical journal

Duncan Ayers¹  Philip JR Day¹ 

关键词: RNAi;    siRNA;    nanoparticles;    gene silencing;    delivery;   

DOI  :  

学科分类：医学（综合）

来源: University of Malta * Medical School

【 摘 要 】

The existence of an intrinsic biochemical pathway enabling specified regulation of gene expression was unheard of until the final years of the last decade. The identificationof ribonucleic acid interference (RNAi) in mammalian cells has nowadays become of extreme importance in the field offunctional genomics and translational medicine. The advent of RNAi technology has brought to the scientific research and pharmaceutical communities the ability to regulate expression ofany desired gene in a reproducible manner. Consequently, such technology may be utilised in the design of novel therapeutics for clinical conditions having dys-regulated gene expression.Since most RNAi-based therapies in the drug development pipeline of pharmaceutical companies utilise short interfering RNA (siRNA), this review will focus on the role of siRNA indrug development.

【 授权许可】

Unknown