title: Dissecting microRNA dysregulation in age-related macular degeneration: new targets for eye gene therapy.
abstract: MicroRNAs (miRNAs) are key regulators of gene expression in humans. Overexpression or depletion of individual miRNAs is associated with human disease. Current knowledge suggests that the retina is influenced by miRNAs and that dysregulation of miRNAs as well as alterations in components of the miRNA biogenesis machinery are involved in retinal diseases, including age-related macular degeneration (AMD). Furthermore, recent studies have indicated that the vitreous has a specific panel of circulating miRNAs and that this panel varies according to the specific pathological stress experienced by the retinal cells. MicroRNA (miRNA) profiling indicates subtype-specific miRNA profiles for late-stage AMD highlighting the importance of proper miRNA regulation in AMD. This review will describe the function of important miRNAs involved in inflammation, oxidative stress and pathological neovascularization, the key molecular mechanisms leading to AMD, and focus on dysregulated miRNAs as potential therapeutic targets in AMD.
authors.affiliation: Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
authors.forename: Anne Louise
authors.initials: AL
authors.lastname: Askou
authors.affiliation: Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
authors.forename: Sidsel
authors.initials: S
authors.lastname: Alsing
authors.affiliation: Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
authors.forename: Andreas
authors.initials: A
authors.lastname: Holmgaard
authors.affiliation: Department of Ophthalmology, Aarhus University Hospital, Aarhus C, Denmark.
authors.forename: Toke
authors.initials: T
authors.lastname: Bek
authors.affiliation: Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
authors.forename: Thomas J
authors.initials: TJ
authors.lastname: Corydon
mesh_terms: D020869:Gene Expression Profiling / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D035683:MicroRNAs / Q000235:genetics* / Q000378:metabolism; D055213:Retinal Pigment Epithelium / Q000378:metabolism / Q000473:pathology*; D057135:Wet Macular Degeneration / Q000235:genetics / Q000378:metabolism / Q000628:therapy
chemical_list: D035683:MicroRNAs
keywords: age-related macular degeneration; gene regulation; gene therapy; inflammation; microRNA; neovascularization; oxidative stress; therapeutic targets