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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 articlepeer-review

Abstract

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
Volume16
Issue number4
DOIs
StatePublished - Dec 2003

Funding

This work was supported by a grant from the Foundation Fighting Blindness, Inc., and Research to Prevent Blindness.

Funders
Foundation Fighting Blindness
Research to Prevent Blindness

    ASJC Scopus subject areas

    • Ophthalmology

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