title: Retinoids and Retinal Diseases.
abstract: Recent progress in molecular understanding of the retinoid cycle in mammalian retina stems from painstaking biochemical reconstitution studies supported by natural or engineered animal models with known genetic lesions and studies of humans with specific genetic blinding diseases. Structural and membrane biology have been used to detect critical retinal enzymes and proteins and their substrates and ligands, placing them in a cellular context. These studies have been supplemented by analytical chemistry methods that have identified small molecules by their spectral characteristics, often in conjunction with the evaluation of models of animal retinal disease. It is from this background that rational therapeutic interventions to correct genetic defects or environmental insults are identified. Thus, most presently accepted modulators of the retinoid cycle already have demonstrated promising results in animal models of retinal degeneration. These encouraging signs indicate that some human blinding diseases can be alleviated by pharmacological interventions.
authors.affiliation: Department of Pharmacology, Cleveland Center for Membrane and Structural Biology, School of Medicine, Case Western Reserve University, Cleveland, Ohio 44106 ; Louis Stokes Cleveland VA Medical Center, Cleveland, Ohio 44106.
authors.forename: Philip D
authors.initials: PD
authors.lastname: Kiser
authors.affiliation: Department of Pharmacology, Cleveland Center for Membrane and Structural Biology, School of Medicine, Case Western Reserve University, Cleveland, Ohio 44106.
authors.forename: Krzysztof
authors.initials: K
authors.lastname: Palczewski
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D006801:Humans; D012164:Retinal Diseases / Q000188:drug therapy / Q000378:metabolism*; D012176:Retinoids / Q000737:chemistry / Q000502:physiology* / Q000627:therapeutic use
chemical_list: D012176:Retinoids
keywords: A2E; LRAT; RPE65; age-related macular degeneration; cone pigments; retinoids; retinol dehydrogenases; rhodopsin; visual cycle; visual cycle modulator