Progress on ocular siRNA gene‐silencing therapy and drug delivery systems

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. A...

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Published inFundamental & clinical pharmacology Vol. 35; no. 1; pp. 4 - 24
Main Authors Jiang, Jinjin, Zhang, Xinru, Tang, Yue, Li, Shuhan, Chen, Jing
Format Journal Article
LanguageEnglish
Published England Wiley Subscription Services, Inc 01.02.2021
Subjects
age‐related macular degeneration
Blindness
Drug delivery
Drug delivery systems
Drugs
Eye diseases
Gene expression
Glaucoma
Immunogenicity
Macular degeneration
non‐viral vectors
Pharmacology
RNA interference technology
RNA-mediated interference
siRNA
siRNA drugs
Vectors (Biology)
siRNA drugs
age-related macular degeneration
glaucoma
RNA interference technology
non-viral vectors
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Summary: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.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-3
content type line 23
ObjectType-Review-1
ISSN:0767-3981
1472-8206
DOI:10.1111/fcp.12561