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Gene-Based Therapies for Leber Hereditary Optic Neuropathy. Hype or Hope?


Mackey, DA and Kearns, LS and Hewitt, AW, Gene-Based Therapies for Leber Hereditary Optic Neuropathy. Hype or Hope?, Asia-Pacific Journal of Ophthalmology, 5, (4) pp. 253-255. ISSN 0129-1653 (2016) [Refereed Article]

Copyright Statement

Copyright 2016 Asia Pacific Academy of Ophthalmology

DOI: doi:10.1097/APO.0000000000000220


Leber hereditary optic neuropathy has now joined Leber congenital amaurosis in the list of genetic eye diseases undergoing gene therapy clinical trials. Although a dramatic response to treatment would be welcome, a minor improvement in vision is a major challenge in efficacy assessment, given this may occur spontaneously as part of the natural history of minor recovery in some patients. Thus, we must await the outcome of adequately powered clinical trials to know if the treatment is effective, particularly given the likely high cost of such therapeutic interventions in the future. We need global cooperation to ensure that the most suitable patients are enrolled in these trials and that support is provided for participants who need to travel from the Asia-Pacific region to Europe or North America if there are no local arms of these trials.

Item Details

Item Type:Refereed Article
Keywords:LHON, AAV gene therapy
Research Division:Biomedical and Clinical Sciences
Research Group:Ophthalmology and optometry
Research Field:Ophthalmology
Objective Division:Health
Objective Group:Clinical health
Objective Field:Clinical health not elsewhere classified
UTAS Author:Mackey, DA (Professor David Mackey)
UTAS Author:Hewitt, AW (Professor Alex Hewitt)
ID Code:111448
Year Published:2016
Web of Science® Times Cited:2
Deposited By:Menzies Institute for Medical Research
Deposited On:2016-09-14
Last Modified:2022-08-25

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