PDF(Pubmed)
Abstract：
While significant progress has been made in understanding and applying gene silencing mechanisms and the treatment of human diseases, there have been still several obstacles in therapeutic use. For the first time, ONPATTRO, as the first small interfering RNA (siRNA) based drug was invented in 2018 for treatment of hTTR with polyneuropathy. Additionally, four other siRNA based drugs naming Givosiran, Inclisiran, Lumasiran, and Vutrisiran have been approved by the US Food and Drug Administration and the European Medicines Agency for clinical use by hitherto. In this review, we have discussed the key and promising advances in the development of siRNA-based drugs in preclinical and clinical stages, the impact of these molecules in bacterial and viral infection diseases, delivery system issues, the impact of administration methods, limitations of siRNA application and how to overcome them and a glimpse into future developments.
摘要：
尽管在理解和应用基因沉默机制以及人类疾病的治疗方面取得了重大进展，在治疗应用中仍然存在一些障碍。第一次,ONPATTRO,2018年发明了第一种基于小干扰RNA（siRNA）的药物，用于治疗多发性神经病的hTTR。此外,另外四种基于siRNA的药物命名为吉沃西兰，Inclisiran,Lumasiran,迄今为止，Vutrisiran和Vutrisiran已被美国食品和药物管理局和欧洲药品管理局批准用于临床使用。在这次审查中,我们已经讨论了基于siRNA的药物在临床前和临床阶段的关键和有希望的进展，这些分子在细菌和病毒感染疾病中的影响，交付系统问题，管理方法的影响，siRNA应用的局限性以及如何克服它们以及对未来发展的一瞥。
