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Therapeutic strategies for Leber's hereditary optic neuropathy: A current update


Gueven, N and Faldu, D, Therapeutic strategies for Leber's hereditary optic neuropathy: A current update, Intractable & Rare Diseases Research., 2, (4) pp. 130-135. ISSN 2186-3644 (2013) [Substantial Review]

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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:Leber's Hereditary Optic Neuropathy, mitochondrial disease, idebenone
Research Division:Biomedical and Clinical Sciences
Research Group:Pharmacology and pharmaceutical sciences
Research Field:Basic pharmacology
Objective Division:Expanding Knowledge
Objective Group:Expanding knowledge
Objective Field:Expanding knowledge in the biological sciences
UTAS Author:Gueven, N (Dr Nuri Guven)
UTAS Author:Faldu, D (Mr Dharmesh Faldu)
ID Code:89462
Year Published:2013
Deposited By:Pharmacy
Deposited On:2014-03-05
Last Modified:2014-03-12

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