Treatment of retinal and choroidal degenerations and dystrophies: Current status and prospects for gene-based therapy

Richard G. Weleber, Daryl E. Kurz, Karmen M. Trzupek

Research output: Contribution to journalReview article

21 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)583-593
Number of pages11
JournalOphthalmology Clinics of North America
Issue number4
StatePublished - Dec 2003


ASJC Scopus subject areas

  • Ophthalmology

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