RNA Interference: Mechanisms, Technical Challenges, and Therapeutic Opportunities
RNA Interference: Mechanisms, Technical Challenges, and Therapeutic Opportunities
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DOI：
10.1007/978-1-4939-1538-5_1
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发表时间：
2015-01-01
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影响因子：
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通讯作者：
Sioud, Mouldy
中科院分区：
文献类型：
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作者：
Sioud, Mouldy
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机制的最新理解已经改进了具有上级效力和特异性的功能性小干扰（si）RNA的设计。关于递送，递送策略的新发展促进了临床前和临床siRNA应用。这篇综述为指导治疗性siRNA的设计和递送提供了有价值的见解。
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 specifi city. 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.