microRNA治疗在癌症及其他疾病中的研究进展*

microRNAs(miRNAs)是一类内源性、非编码小分子RNAs(约22 nt),在基因表达调控中发挥关键作用。已有研究表明,miRNAs失调是造成多种人类疾病的原因,如癌症、病毒感染及自身免疫性疾病等。补充或抑制miRNAs功能与活性已成为多种疾病治疗的新策略,抗肿瘤miR-34 mimics、治疗HCV感染的anti-miR-122等基于miRNAs的治疗方案已进入临床试验。重点就miRNAs治疗在癌症及其他疾病中的最新研究进展进行综述,并对目前开发安全有效miRNAs治疗策略所面临的挑战进行分析。

Advance of microRNA Therapeutics in Cancer and Other Diseases

microRNAs (miRNAs) are a class of endogenous, small non-coding RNAs (about 22 nt) that have critical roles in gene expression. Functional studies have identified miRNAs dysregulation as a cause in many human diseases, including cancer, viral infection, and autoimmune disorders. Restoring or suppressing miRNAs function and activity has become an attractive therapeutic method for the management of cancer and other diseases. Several miRNA-based therapeutics have reached clinical development, including a mimic of the tumor suppressor miR-34 for treating cancer and anti-miRs targeted miR-122 for treating HCV infection. In this review, we summarize the recent advances in our knowledge of miRNA therapeutics in cancer and other diseases and discuss ongoing challenges to ensure the safety and efficacy of miRNA therapeutics in vivo.