Treatment of retinal and choroidal degenerations and dystrophies: current status and prospects for gene-based therapy.
Inherited retinal and choroidal degenerations account for a significant portion of blindness in children and young adults. This article reviews the current status and future prospects for the treatment of these disorders. Current treatment strategies include nutritional intervention for gyrate atrophy of the choroid and retina with hyperornithinemia, abetalipoproteinemia, and Refsum's disease, as well as vitamin A supplementation for retinitis pigmentosa. Future therapeutic prospects include gene therapy for both recessive and dominant disease, secondary gene-based therapies, such as pharmaceutic gene product replacement and treatment with survival factors, anti-apoptotic agents, and calcium blockers, and, finally, stem cell therapy.
Weleber
Richard G
RG
Casey Eye Institute, Department of Ophthalmology, Oregon Health & Science University, 3375 Southwest Terwilliger Boulevard, Portland, OR 97239, USA. weleberr@ohsu.edu
Kurz
Daryl E
DE
Trzupek
Karmen M
KM
D000818:Animals; D015862:Choroid Diseases / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D012162:Retinal Degeneration / Q000628:therapy*