Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine.
Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine.
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DOI：
10.1016/j.omtn.2017.06.005
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发表时间：
2017-09-15
期刊：
影响因子：
--
通讯作者：
Lee SS
中科院分区：
文献类型：
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作者：
Chakraborty C;Sharma AR;Sharma G;Doss CGP;Lee SS
在过去的几年中，治疗性microRNA（miRNA）和小干扰RNA（siRNA）是商业空间中作为未来药物的一些最重要的生物药物。这篇综述总结了基于miRNA和siRNA的新药的专利，并提供了有关正在投资miRNA和siRNA分子的治疗开发的重要生物制药公司的快照。对单个siRNA和miRNA药物的深入见解已经描述了它们的现状，这在治疗景观中越来越受到关注。生物制药的努力与他们的临床前和/或临床试验的状态进行了讨论。在这里，一些挫折已经强调了在生物制药开发的miRNA和siRNA作为个体疗法。最后，简要介绍了药物动力学、药效学与吸收、分布、代谢和排泄（ADME），这是这些疗法的基本发展过程，以及基于miRNA和siRNA的药物的递送系统。
In the past few years, therapeutic microRNA (miRNA) and small interfering RNA (siRNA) are some of the most important biopharmaceuticals that are in commercial space as future medicines. This review summarizes the patents of miRNA- and siRNA-based new drugs, and also provides a snapshot about significant biopharmaceutical companies that are investing for the therapeutic development of miRNA and siRNA molecules. An insightful view about individual siRNA and miRNA drugs has been depicted with their present status, which is gaining attention in the therapeutic landscape. The efforts of the biopharmaceuticals are discussed with the status of their preclinical and/or clinical trials. Here, some of the setbacks have been highlighted during the biopharmaceutical development of miRNA and siRNA as individual therapeutics. Finally, a snapshot is illustrated about pharmacokinetics, pharmacodynamics with absorption, distribution, metabolism, and excretion (ADME), which is the fundamental development process of these therapeutics, as well as the delivery system for miRNA- and siRNA-based drugs.