RNAi 介质的递送。
Delivery of RNAi mediators.
机构信息
Bioo Scientific, 3913 Todd Lane Suite 312, Austin, TX 78744, USA.
出版信息
Wiley Interdiscip Rev RNA. 2010 Sep-Oct;1(2):341-50. doi: 10.1002/wrna.12. Epub 2010 Jun 3.
Abstract
Delivering polynucleotides into animals has been a major challenge facing their success as therapeutic agents. Given the matured understanding of antibody-mediated delivery techniques, it is possible to rationally design delivery vehicles that circulate in the blood stream and are specifically delivered into target organs. If the targeting moiety is designed to contain the cargo of an RNAi mediator without impacting its paratope, directed delivery can be achieved. In this article, we review the state of art in delivery technology for RNA mediators and address how this technique could soon be used to enhance the efficacy of the numerous small RNA therapeutic programs currently under evaluation.
摘要
将多核苷酸递送到动物体内一直是其作为治疗剂取得成功的主要挑战。鉴于对抗体介导的递药技术的成熟理解，有可能合理设计在血液中循环并专门递送到靶器官的递药载体。如果靶向部分被设计成包含 RNAi 介导物的货物而不影响其变构位，则可以实现靶向递药。本文综述了 RNA 介导物递药技术的最新进展，并探讨了该技术如何很快用于提高目前正在评估的众多小 RNA 治疗方案的疗效。
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