Small-interfering RNAs (siRNAs) as a promising tool for ocular therapy

被引:107
作者
Guzman-Aranguez, A. [1 ]
Loma, P. [1 ]
Pintor, J. [1 ]
机构
[1] Univ Complutense Madrid, Fac Opt & Optometry, Dept Biochem & Mol Biol 4, Madrid 28037, Spain
关键词
siRNAs; therapy; eye; glaucoma; retinitis pigmentosa; age-related macular degeneration; diabetic retinopathy; corneal neovascularization; TISSUE GROWTH-FACTOR; TRABECULAR MESHWORK CELLS; AQUEOUS-HUMOR DYNAMICS; DOUBLE-STRANDED-RNA; EXPRESSION IN-VIVO; EXTRACELLULAR-MATRIX; INTRAOCULAR-PRESSURE; CORNEAL NEOVASCULARIZATION; RETINITIS-PIGMENTOSA; RETINAL DEGENERATION;
D O I
10.1111/bph.12330
中图分类号
R9 [药学];
学科分类号
100702 [药剂学];
摘要
RNA interference (RNAi) can be used to inhibit the expression of specific genes in vitro and in vivo, thereby providing an extremely useful tool for investigating gene function. Progress in the understanding of RNAi-based mechanisms has opened up new perspectives in therapeutics for the treatment of several diseases including ocular disorders. The eye is currently considered a good target for RNAi therapy mainly because it is a confined compartment and, therefore, enables local delivery of small-interfering RNAs (siRNAs) by topical instillation or direct injection. However, delivery strategies that protect the siRNAs from degradation and are suitable for long-term treatment would be help to improve the efficacy of RNAi-based therapies for ocular pathologies. siRNAs targeting critical molecules involved in the pathogenesis of glaucoma, retinitis pigmentosa and neovascular eye diseases (age-related macular degeneration, diabetic retinopathy and corneal neovascularization) have been tested in experimental animal models, and clinical trials have been conducted with some of them. This review provides an update on the progress of RNAi in ocular therapeutics, discussing the advantages and drawbacks of RNAi-based therapeutics compared to previous treatments.
引用
收藏
页码:730 / 747
页数:18
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