Age‐related macular degeneration (AMD) and glaucoma are global ocular diseases with high blindness rate. RNA interference (RNAi) is being increasingly used in the treatment of these disorders with siRNA drugs, bevasiranib, AGN211745 and PF‐04523655 for AMD, and SYL040012 and QPI‐1007 for glaucoma. Administration routes and vectors of gene drugs affect their therapeutic effect. Compared with the non‐viral vectors, viral vectors have limited payload capacity and potential immunogenicity. This review summarizes the progress of the ocular siRNA gene‐silencing therapy by focusing on siRNA drugs for AMD and glaucoma already used in clinical research, the main routes of drug delivery and the non‐viral vectors for siRNA drugs.

中文翻译：

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眼部siRNA基因沉默治疗及给药系统研究进展

年龄相关性黄斑变性 (AMD) 和青光眼是全球眼科疾病，失明率高。RNA 干扰 (RNAi) 越来越多地用于治疗这些疾病，包括 siRNA 药物，bevasiranib、AGN211745 和 PF-04523655 用于治疗 AMD，SYL040012 和 QPI-1007 用于治疗青光眼。基因药物的给药途径和载体影响其治疗效果。与非病毒载体相比，病毒载体的有效载荷能力和潜在的免疫原性有限。本综述主要针对临床研究中已使用的治疗AMD和青光眼的siRNA药物、主要给药途径和siRNA药物的非病毒载体，总结眼部siRNA基因沉默治疗的进展。