Sioud Mouldy
Department of Immunology, Institute for Cancer Research, Oslo University Hospital, Montebello, 0310, Oslo, Norway,
Methods Mol Biol. 2015;1218:1-15. doi: 10.1007/978-1-4939-1538-5_1.
The ability to inhibit gene expression via RNA interference (RNAi) has a broad therapeutic potential for various human diseases such as infections and cancers. Recent advances in mechanistic understanding of RNAi have improved the design of functional small interfering (si) RNAs with superior potency and specificity. With respect to delivery, new developments in delivery strategies have facilitated preclinical and clinical siRNA applications. This review provides valuable insights to guide the design and delivery of therapeutic siRNAs.
通过RNA干扰（RNAi）抑制基因表达的能力对于各种人类疾病，如感染和癌症，具有广泛的治疗潜力。对RNAi机制理解的最新进展改进了具有更高效力和特异性的功能性小干扰（si）RNA的设计。在递送方面，递送策略的新进展促进了临床前和临床siRNA的应用。本综述为指导治疗性siRNA的设计和递送提供了有价值的见解。
