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

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.

• Authors: Jinjin Jiang, Xinru Zhang, Yue Tang, Shuhan Li, Jing Chen
• Journal: Fundamental & clinical pharmacology (Fundam Clin Pharmacol) Vol. 35 Issue 1 Pg. 4-24 (Feb 2021) ISSN: 1472-8206 [Electronic] England
• PMID: 32298491 (Publication Type: Journal Article, Review)
• Copyright: © 2020 Société Française de Pharmacologie et de Thérapeutique.

Chemical References

• RNA, Small Interfering

Topics

• Blood-Retinal Barrier
• Drug Delivery Systems
• Gene Silencing
• Genetic Therapy (methods)
• Glaucoma (therapy)
• Macular Degeneration (therapy)
• RNA, Small Interfering (administration & dosage, genetics)