Inherited diseases of photoreceptors and prospects for gene therapy

Pharmacogenomics. 2008 Mar;9(3):335-47. doi: 10.2217/14622416.9.3.335.

Abstract

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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans
  • Photoreceptor Cells, Vertebrate / pathology*
  • Retina / anatomy & histology
  • Retina / pathology
  • Retinal Diseases / genetics*
  • Retinal Diseases / therapy*