Genetic retinal diseases

Purpose To describe the current status of treatment for inherited retinal disease, and illustrate the challenges that lie ahead. Methods An overview of current treatment trials for inherited retinal disease in humans and animals will be used to illustrate where success has been achieved, but also wh...

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Bibliographic Details
Published inActa ophthalmologica (Oxford, England) Vol. 90; no. s249
Main Author LEROY, BP
Format Journal Article
LanguageEnglish
Published Oxford, UK Blackwell Publishing Ltd 01.09.2012
Wiley Subscription Services, Inc
Subjects
Gene therapy
Medical research
Ophthalmology
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Summary:Purpose To describe the current status of treatment for inherited retinal disease, and illustrate the challenges that lie ahead. Methods An overview of current treatment trials for inherited retinal disease in humans and animals will be used to illustrate where success has been achieved, but also where challenges remain. These include gene therapy trials in RPE65‐related Leber congenital amaurosis, Stargardt macular dystrophy and choroideraemia. Results Albeit that encouraging breakthroughs have been made with regard to treating inherited retinal diseases, such as in the gene therapy trials in RPE65‐related Leber congenital amaurosis, challenges lie ahead, such as gene‐specificity of gene therapy, intravitreal as opposed to subretinal injections, stem cell control, and cost. Conclusion Despite initial success of trials in inherited retinal diseases, quite a number of obstacles remain.
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ISSN:1755-375X
1755-3768
DOI:10.1111/j.1755-3768.2012.4213.x