a Faculty of Pharmaceutical Sciences , Hokkaido University , Sapporo , Japan.
b Faculty of Pharmacy , Assiut University , Assiut , Egypt.
Expert Opin Drug Deliv. 2018 Nov;15(11):1053-1065. doi: 10.1080/17425247.2018.1520836. Epub 2018 Sep 25.
The discovery of RNA interference (RNAi) earned the 2006 Nobel Prize in Physiology or Medicine for its biological significance and potential for developing novel therapeutics. The small interfering RNA (siRNA) is the most promising tool for translating RNAi to clinical use. Efforts are ongoing to improve siRNA delivery through developing novel biomaterials and delivery strategies. Given time, it appears that siRNA drugs will eventually become a reality.
The currently used approaches for siRNA delivery are discussed with a focus on siRNA therapeutics currently in clinical testing. A comparison of advantageous aspects of currently available platforms and the possibility of further optimization for increased efficiency and safety are presented. Future directions in siRNA delivery are also highlighted.
The recent success in the field of siRNA delivery is based mainly on developing new biomaterials with extraordinarily high activities. Notably, the introduction of ionizable lipids and novel targeting ligands represent two huge steps for realizing siRNA therapy. The currently available systems are largely directed to the liver and the new challenge is to extend their applicability for treating diseases of other organs. Active targeting to different organs is the most promising approach for developing new siRNA-based therapeutics.
RNA 干扰（RNAi）的发现因其生物学意义和开发新型疗法的潜力而获得 2006 年诺贝尔生理学或医学奖。小干扰 RNA（siRNA）是将 RNAi 转化为临床应用最有前途的工具。人们正在努力通过开发新型生物材料和输送策略来改进 siRNA 的输送。假以时日，siRNA 药物似乎最终将成为现实。
本文讨论了目前用于 siRNA 输送的方法，重点是目前正在临床测试的 siRNA 疗法。比较了现有平台的有利方面，并提出了进一步优化以提高效率和安全性的可能性。还强调了 siRNA 输送的未来方向。
siRNA 输送领域的近期成功主要基于开发具有极高活性的新型生物材料。值得注意的是，可离子化脂质和新型靶向配体的引入代表了实现 siRNA 治疗的两个巨大步骤。目前可用的系统主要针对肝脏，新的挑战是将其适用性扩展到治疗其他器官的疾病。针对不同器官的主动靶向是开发新型基于 siRNA 的治疗方法的最有前途的方法。
