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1 General aspects (0)   1.1 Epidemiology (7)   1.2 Population genetics (1)   1.3 Pathogenesis (8)   1.4 Quality of life (5)   1.5 Glaucomas as cause of blindness (3)   1.6 Prevention and screening (14) 2 Anatomical structures in glaucoma (0)   2.1 Conjunctiva (10)   2.2 Cornea (17)   2.3 Sclera (8)   2.4 Anterior chamber angle (8)   2.5 Meshwork (0)     2.5.1 Trabecular meshwork (12)     2.5.2 Schlemms canal (1)     2.5.3 Scleral outlet channels (0)   2.6 Aqueous humor dynamics (0)     2.6.1 Production (0)     2.6.2 Outflow (0)       2.6.2.1 Trabecular meshwork (2)       2.6.2.2 Uveoscleral (2)     2.6.3 Compostion (5)   2.7 Episcleral veins and venous pressure (0)   2.8 Iris (7)   2.9 Ciliary body (1)   2.10 Lens (0)   2.11 Vitreous body (1)   2.12 Choroid, peripapillary choroid, peripapillary atrophy (5)   2.13 Retina and retinal nerve fibre layer (42)   2.14 Optic disc (26)   2.15 Optic nerve (2)   2.16 Chiasma and retrochiasmal central nervous system (7)   2.17 Stem cells (2)   2.20 Other (0) 3 Laboratory methods (0)   3.1 Microscopy (3)   3.2 Electron microscopy (0)   3.3 Immunohistochemistry (4)   3.4 Molecular genetics (0)     3.4.1 Linkage studies (0)     3.4.2 Gene studies (40)   3.5 Molecular biology incl. 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Miscellaneous (17)

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Abstract #54562 Published in IGR 15-3

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

Guzman-Aranguez A; Loma P; Pintor J
British Journal of Pharmacology 2013; 170: 730-747

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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.

Department of Biochemistry and Molecular Biology IV, Faculty of Optics and Optometry, Universidad Complutense de Madrid, Madrid, Spain.

Full article

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Classification:

3.5 Molecular biology incl. SiRNA (Part of: 3 Laboratory methods)

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