Shanxi Medical University, No. 56 Xinjian South Road, Yingze District, Taiyuan, China.
Queen Mary School, Nanchang University, Nanchang, China.
Mol Diagn Ther. 2018 Oct;22(5):551-569. doi: 10.1007/s40291-018-0338-8.
Small interfering RNAs (siRNAs) are an attractive new agent with potential as a therapeutic tool because of its ability to inhibit specific genes for many conditions, including viral infections and cancers. However, despite this potential, many challenges remain, including off-target effects, difficulties with delivery, immune responses, and toxicity. Traditional genetic vectors do not guarantee that siRNAs will silence genes in vivo. Rational design strategies, such as chemical modification, viral vectors, and non-viral vectors, including cationic liposomes, polymers, nanocarriers, and bioconjugated siRNAs, provide important opportunities to overcome these challenges. We summarize the results of research into vector delivery of siRNAs as a therapeutic agent from their design to clinical trials in ophthalmic diseases, cancers, respiratory diseases, and liver virus infections. Finally, we discuss the current state of siRNA delivery methods and the need for greater understanding of the requirements.
小干扰 RNA（siRNA）是一种有吸引力的新型药物，具有作为治疗工具的潜力，因为它能够抑制许多疾病（包括病毒感染和癌症）的特定基因。然而，尽管有这种潜力，仍然存在许多挑战，包括脱靶效应、递药困难、免疫反应和毒性。传统的基因载体并不能保证 siRNA 在体内沉默基因。合理的设计策略，如化学修饰、病毒载体和非病毒载体，包括阳离子脂质体、聚合物、纳米载体和生物偶联的 siRNA，为克服这些挑战提供了重要的机会。我们总结了 siRNA 作为治疗剂的载体传递研究结果，从设计到眼科疾病、癌症、呼吸道疾病和肝脏病毒感染的临床试验。最后，我们讨论了 siRNA 递药方法的现状以及对更深入了解需求的必要性。
