John Curtin School of Medical Research, The Australian National University, Canberra, ACT, Australia.
ANU Medical School, The Australian National University, Canberra, ACT, Australia.
Adv Exp Med Biol. 2018;1074:37-43. doi: 10.1007/978-3-319-75402-4_5.
MicroRNA (miRNA) are a class of endogenously expressed small non-coding RNA molecules that function by repressing or silencing post-transcriptional gene expression. While miRNAs were only identified in humans as recently as the turn of this century, some miRNA-based agents are already in Phase 2 clinical trials (Christopher et al. 2016). This rapid progress from initial discovery to drug development reflects the effectiveness of miRNAs as therapeutic targets. Further, their use as therapeutic agents in the treatment of diseases such as Alzheimer's disease (Wang et al. 2014) supports their use in other neurodegenerative diseases, such as Age-Related Macular Degeneration (AMD). However, despite ∼300 miRNAs reportedly expressed in the human retina (Xu 2009), relatively little research has been conducted into the therapeutic potential of miRNAs for the treatment of AMD. This review will investigate the use of miRNAs as therapeutic and diagnostic molecules for AMD.
微小 RNA（miRNA）是一类内源性表达的小非编码 RNA 分子，通过抑制或沉默转录后基因表达来发挥作用。虽然 miRNA 直到本世纪初才在人类中被发现，但一些基于 miRNA 的药物已经进入了 2 期临床试验阶段（Christopher 等人，2016）。从最初的发现到药物开发的快速进展反映了 miRNA 作为治疗靶点的有效性。此外，它们作为治疗剂在治疗阿尔茨海默病（Wang 等人，2014）等疾病中的应用支持了它们在其他神经退行性疾病中的应用，如年龄相关性黄斑变性（AMD）。然而，尽管据报道在人类视网膜中表达了约 300 种 miRNA（Xu 2009），但对于 miRNA 治疗 AMD 的治疗潜力的研究相对较少。这篇综述将探讨 miRNA 作为 AMD 的治疗和诊断分子的应用。
