Leber hereditary optic neuropathy

Common Name(s)

Leber hereditary optic neuropathy, Leber's optic atrophy, Leber's Disease

Leber hereditary optic neuropathy (LHON) is an inherited form of vision loss. Although this condition usually begins in a person's teens or twenties, rare cases may appear in early childhood or later in adulthood. For unknown reasons, males are affected much more often than females. This condition is caused by mutations in the MT-ND1, MT-ND4, MT-ND4L, and MT-ND6 genes.
 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Leber hereditary optic neuropathy" for support, advocacy or research.

United Mitochondrial Disease Foundation

The United Mitochondrial Disease Foundation strives to promote research and education for the diagnosis, treatment and cures of mitochondrial disorders and to provide support to affected individuals and families.

Last Updated: 28 Jul 2015

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Leber hereditary optic neuropathy" for support, advocacy or research.

United Mitochondrial Disease Foundation

The United Mitochondrial Disease Foundation strives to promote research and education for the diagnosis, treatment and cures of mitochondrial disorders and to provide support to affected individuals and families.

http://www.umdf.org

Last Updated: 28 Jul 2015

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Leber hereditary optic neuropathy" returned 82 free, full-text research articles on human participants. First 3 results:

Gene Therapy for Leber Hereditary Optic Neuropathy: Initial Results.
 

Author(s): William J Feuer, Joyce C Schiffman, Janet L Davis, Vittorio Porciatti, Phillip Gonzalez, Rajeshwari D Koilkonda, Huijun Yuan, Anil Lalwani, Byron L Lam, John Guy

Journal: Ophthalmology. 2016 Mar;123(3):558-70.

 

Leber hereditary optic neuropathy (LHON) is a disorder characterized by severe and rapidly progressive visual loss when caused by a mutation in the mitochondrial gene encoding NADH:ubiquinone oxidoreductase subunit 4 (ND4). We have initiated a gene therapy trial to determine the safety ...

Last Updated: 23 Feb 2016

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Measurement of Systemic Mitochondrial Function in Advanced Primary Open-Angle Glaucoma and Leber Hereditary Optic Neuropathy.
 

Author(s): Nicole J Van Bergen, Jonathan G Crowston, Jamie E Craig, Kathryn P Burdon, Lisa S Kearns, Shiwani Sharma, Alex W Hewitt, David A Mackey, Ian A Trounce

Journal:

 

Primary Open Angle Glaucoma (POAG) is a common neurodegenerative disease characterized by the selective and gradual loss of retinal ganglion cells (RGCs). Aging and increased intraocular pressure (IOP) are glaucoma risk factors; nevertheless patients deteriorate at all levels of IOP, ...

Last Updated: 27 Oct 2015

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Trial end points and natural history in patients with G11778A Leber hereditary optic neuropathy : preparation for gene therapy clinical trial.
 

Author(s): Byron L Lam, William J Feuer, Joyce C Schiffman, Vittorio Porciatti, Ruth Vandenbroucke, Potyra R Rosa, Giovanni Gregori, John Guy

Journal: JAMA Ophthalmol. 2014 Apr;132(4):428-36.

 

IMPORTANCE Establishing the natural history of G11778A Leber hereditary optic neuropathy (LHON) is important to determine the optimal end points to assess the safety and efficacy of a planned gene therapy trial. OBJECTIVE To use the results of the present natural history study of ...

Last Updated: 11 Apr 2014

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Leber hereditary optic neuropathy" returned 8 free, full-text review articles on human participants. First 3 results:

Leber hereditary optic neuropathy: bridging the translational gap.
 

Author(s): Neringa Jurkute, Patrick Yu-Wai-Man

Journal: Curr Opin Ophthalmol. 2017 Sep;28(5):403-409.

 

Leber hereditary optic neuropathy (LHON) is the most common primary mitochondrial DNA (mtDNA) genetic disorder in the population. We address the clinical evolution of the disease, the secondary etiological factors that could contribute to visual loss, and the challenging task of developing ...

Last Updated: 26 Jun 2017

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

Author(s): David A Mackey, Lisa S Kearns, Alex W Hewitt

Journal: Asia Pac J Ophthalmol (Phila). ;5(4):253-5.

 

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, ...

Last Updated: 4 Aug 2016

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Is there treatment for Leber hereditary optic neuropathy?
 

Author(s): Jason H Peragallo, Nancy J Newman

Journal: Curr Opin Ophthalmol. 2015 Nov;26(6):450-7.

 

To discuss recent advances in potential treatments for Leber hereditary optic neuropathy (LHON), a typically visually devastating hereditary optic neuropathy caused by mutations in the mitochondrial genome.

Last Updated: 9 Oct 2015

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Clinical Trial Information This information is provided by ClinicalTrials.gov

Safety and Efficacy Study of Gene Therapy for The Treatment of Leber's Hereditary Optic Neuropathy
 

Status: Recruiting

Condition Summary: Leber Hereditary Optic Neuropathy

 

Last Updated: 2 Jul 2017

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Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
 

Status: Recruiting

Condition Summary: Leber's Hereditary Optic Neuropathy

 

Last Updated: 10 Jan 2017

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Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
 

Status: Recruiting

Condition Summary: Leber's Hereditary Optic Neuropathy (LHON)

 

Last Updated: 1 Mar 2017

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