Current gene therapy trials for inherited retinal disorders

Summary Purpose To discuss the current status of gene therapy for inherited retinal disease. Methods Data from the literature on gene therapy trials for several inherited retinopathies will be combined with those of the Phase 1, Phase 1 follow‐on and Phase 3 trials for RPE65‐related inherited retina...

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Bibliographic Details
Published in:Acta ophthalmologica (Oxford, England) England), 2016-10, Vol.94 (S256), p.n/a
Main Authors: Leroy, B.P., Maguire, A.M., Russell, S.R., Wellman, J., Yu, Z.F., Chung, D.C., High, K.A., Bennett, J.
Format: Article
Language:English
Subjects:
Children
Data processing
Disease
Expression vectors
Gene therapy
Ophthalmology
Retina
Retinal degeneration
Stabilization
Visual perception
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Summary:Summary Purpose To discuss the current status of gene therapy for inherited retinal disease. Methods Data from the literature on gene therapy trials for several inherited retinopathies will be combined with those of the Phase 1, Phase 1 follow‐on and Phase 3 trials for RPE65‐related inherited retinal disease at The Children's Hospital of Philadelphia, Philadelphia, PA, USA. Results Gene therapy is capable of improving or stabilizing visual function. According to some recent reports, there is progression of disease in RPE65‐related IRD trial participants despite successful subretinal delivery of RPE65 using an AAV2 vector. Other data suggest that there may at least be a decrease in the speed of retinal degeneration, if not stabilization. Conclusions Gene therapy for inherited retinal disease using viral vectors has demonstrated safety and improvement or stabilization of visual function in some diseases. Whereas disease progression is noted in some RPE65‐related IRD trial participants, despite successful application of subretinal gene therapy, others may even have either a stable or slower disease course after treatment.
ISSN:1755-375X
1755-3768
DOI:10.1111/j.1755-3768.2016.0160