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Idebenone treatment in Leber's hereditary optic neuropathy: rationale and efficacy


Guven, N and Faldu, D, Idebenone treatment in Leber's hereditary optic neuropathy: rationale and efficacy, Expert opinion on orphan drugs, 2, (4) pp. 130-135. ISSN 2167-8707 (2013) [Substantial Review]

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DOI: doi:10.1517/21678707.2013.772894


Leber's hereditary optic neuropathy (LHON) is a rare mitochondrial retinopathy, caused by mutations in subunits of complex I of the respiratory chain, which leads to elevated levels of oxidative stress and an insufficient energy supply. This molecular pathology is thought to be responsible for the dysfunction and eventual apoptotic loss of retinal ganglion cells in the eye, which ultimately results in blindness. Many strategies, ranging from neuroprotectants, antioxidants, anti-apoptotic- and anti-inflammatory compounds have been tested with mixed results. Currently, the most promising compounds are shortchain quinones that have been shown to protect the vision of LHON patients during the early stages of the disease. This commentary gives a brief overview on the current status of tested therapeutics and also addresses future developments such as the use of gene therapy that hopefully will provide safe and efficient therapy options for all LHON patients.

Item Details

Item Type:Substantial Review
Keywords:idebenone Leber's hereditary optic neuropathy LHON
Research Division:Biomedical and Clinical Sciences
Research Group:Pharmacology and pharmaceutical sciences
Research Field:Clinical pharmacology and therapeutics
Objective Division:Expanding Knowledge
Objective Group:Expanding knowledge
Objective Field:Expanding knowledge in the biological sciences
UTAS Author:Guven, N (Dr Nuri Guven)
UTAS Author:Faldu, D (Mr Dharmesh Faldu)
ID Code:83020
Year Published:2013
Web of Science® Times Cited:4
Deposited By:Pharmacy
Deposited On:2013-02-27
Last Modified:2014-05-15
Downloads:1 View Download Statistics

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