The photoreceptor cells of the retina are subject to a wide range of genetic diseases. This review summarizes current knowledge regarding an important group of retinal diseases caused by mutations in photoreceptor-enriched genes. In addition, progress toward treatment of a variety of these diseases in animal models via adeno-associated virus gene therapy is described. Although no human trials have yet been initiated to treat diseases caused by mutations in photoreceptor-enriched genes, there is a great deal of optimism regarding the prospects of treating these diseases using adeno-associated virus gene therapy.

Original languageEnglish
Pages (from-to)335-347
Number of pages13
Issue number3
StatePublished - Mar 2008


  • Adeno-associated virus
  • Gene therapy
  • Photoreceptors
  • Retina
  • cis-regulation element


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