Inherited diseases of photoreceptors and prospects for gene therapy.
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.
Montana
Cynthia L
CL
Department of Pathology and Immunology, Washington University School of Medicine, Campus Box 8118; 660 South Euclid Ave. St. Louis, MO 63110, USA.
Corbo
Joseph C
JC
D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006801:Humans; D020419:Photoreceptor Cells, Vertebrate / Q000473:pathology*; D012160:Retina / Q000033:anatomy & histology / Q000473:pathology; D012164:Retinal Diseases / Q000235:genetics* / Q000628:therapy*