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Progress on ocular siRNA gene‐silencing therapy and drug delivery systems
Author(s) -
Jiang Jinjin,
Zhang Xinru,
Tang Yue,
Li Shuhan,
Chen Jing
Publication year - 2021
Publication title -
fundamental and clinical pharmacology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.655
H-Index - 73
eISSN - 1472-8206
pISSN - 0767-3981
DOI - 10.1111/fcp.12561
Subject(s) - rna interference , gene silencing , glaucoma , small interfering rna , immunogenicity , genetic enhancement , medicine , drug , blindness , macular degeneration , pharmacology , biology , gene , rna , ophthalmology , immunology , optometry , immune system , genetics
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.