眼内 siRNA 基因沉默治疗和药物传递系统的研究进展。

Progress on ocular siRNA gene-silencing therapy and drug delivery systems.

机构信息

State Key Laboratory of Functions and Applications of Medicinal Plants, Guizhou Medical University, No.1, DongQing Road, Guiyang, 550014, People's Republic of China.

Department of Pharmacy, China Pharmaceutical University, No.639, Longmian Avenue, Nanjing, 211198, People's Republic of China.

出版信息

Fundam Clin Pharmacol. 2021 Feb;35(1):4-24. doi: 10.1111/fcp.12561. Epub 2020 May 8.

DOI:10.1111/fcp.12561

PMID:32298491

Abstract

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.

摘要

年龄相关性黄斑变性（AMD）和青光眼是具有高失明率的全球性眼病。RNA 干扰（RNAi）正越来越多地被用于治疗这些疾病，其中包括针对 AMD 的 siRNA 药物贝伐单抗、AGN211745 和 PF-04523655，以及针对青光眼的 SYL040012 和 QPI-1007。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，病毒载体的载物能力有限，且具有潜在的免疫原性。本综述重点介绍了已用于临床研究的 AMD 和青光眼的 siRNA 药物，综述了眼内 siRNA 基因沉默治疗的进展，包括用于 AMD 和青光眼的 siRNA 药物的主要给药途径和非病毒载体。