Inherited Retinal Disorders Service, Massachusetts Eye and Ear, Department of Ophthalmology, Harvard Medical School, Boston, MA, United States.
Semin Ophthalmol. 2021 Aug 18;36(5-6):452-457. doi: 10.1080/08820538.2021.1914116. Epub 2021 May 19.
Antisense oligonucleotides (AON) are synthetic single-stranded fragments of nucleic acids that bind to a specific complementary messenger RNA (mRNA) sequence and change the final gene product. AON were initially approved for treating cytomegalovirus retinitis and have shown promise in treating Mendelian systemic disease. AON are currently being investigated as a treatment modality for many ophthalmic diseases, including inherited retinal disorders (IRD), inflammatory response and wound healing after glaucoma surgery, and macular degeneration. They provide a possible solution to gene therapy for IRD that are not candidates for adeno-associated virus (AAV) delivery. This chapter outlines the historical background of AON and reviews clinical applications and ongoing clinical trials.
反义寡核苷酸（AON）是一种合成的单链核酸片段，可与特定的互补信使 RNA（mRNA）序列结合，并改变最终的基因产物。AON 最初被批准用于治疗巨细胞病毒视网膜炎，并在治疗孟德尔系统疾病方面显示出前景。AON 目前正在作为许多眼科疾病的治疗方法进行研究，包括遗传性视网膜疾病（IRD）、青光眼手术后的炎症反应和伤口愈合以及黄斑变性。它们为不是腺相关病毒（AAV）递送候选物的 IRD 的基因治疗提供了一种可能的解决方案。本章概述了 AON 的历史背景，并回顾了临床应用和正在进行的临床试验。
