攻克疾病：小干扰RNA疗法进展报告
Knocking down disease: a progress report on siRNA therapeutics.
作者信息
Wittrup Anders, Lieberman Judy
机构信息
1] Program in Cellular and Molecular Medicine, Boston Children's Hospital and Department of Pediatrics, Harvard Medical School, Boston, Massachusetts 02115, USA. [2] Department of Clinical Sciences, Section for Oncology and Pathology, Lund University, 221 85 Lund, Sweden.
Program in Cellular and Molecular Medicine, Boston Children's Hospital and Department of Pediatrics, Harvard Medical School, Boston, Massachusetts 02115, USA.
出版信息
Nat Rev Genet. 2015 Sep;16(9):543-52. doi: 10.1038/nrg3978.
Small interfering RNAs (siRNAs), which downregulate gene expression guided by sequence complementarity, can be used therapeutically to block the synthesis of disease-causing proteins. The main obstacle to siRNA drugs - their delivery into the target cell cytosol - has been overcome to allow suppression of liver gene expression. Here, we review the results of recent clinical trials of siRNA therapeutics, which show efficient and durable gene knockdown in the liver, with signs of promising clinical outcomes and little toxicity. We also discuss the barriers to more widespread applications that target tissues besides the liver and the most promising avenues to overcome them.
小干扰RNA（siRNA）可通过序列互补性下调基因表达，可用于治疗性阻断致病蛋白的合成。siRNA药物的主要障碍——将其递送至靶细胞胞质溶胶——已被克服，从而能够抑制肝脏基因表达。在此，我们综述了siRNA疗法近期临床试验的结果，这些结果显示在肝脏中基因敲低高效且持久，有临床结果良好且毒性小的迹象。我们还讨论了除肝脏外针对其他组织更广泛应用的障碍以及克服这些障碍最有前景的途径。
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