[摘要]：Since 2006, when Fire and Mello were awarded the Nobel Prize in Medicine for their work on RNA interference (RNAi), there has been significant progress towards transforming what began as an important laboratory tool for understanding gene function into a drug development platform capable of revolutionizing medicine. Major advances have been made in both local and systemic delivery of small interfering RNAs (siRNAs) in animal models of disease. In particular, novel lipid nano particles and targeted polymer- and cyclodextrin-based particles are providing new ways of delivering siRNAs to liver and to tumors, and these have led to the initiation of a number of clinical trials across various disease areas that are providing useful information regarding safety, drug delivery and pharmacodynamic activity. Oncology is a key testing ground for RNAi therapeutics, as the emergence of new targets that are not druggable by traditional means provides an opportunity for using RNAi-based drug platforms to expand the reach of targeted therapy The recent discovery of activating mutations in GNAQ and GNA11 in the majority of uveal melanoma patients raises the possibility that targeting these genes with systemically delivered siRNA may provide a treatment breakthrough for this devastating disease following spread to the liver
