A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic neuropathy (LHON). The study was conducted in collaboration with the Seoul National University College of ...
Feb 4, 2026
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A study published in Science reveals a cellular mechanism involved in the inheritance of genetic mutations. The study also points to a potential treatment that could reduce the risk of babies being born with serious, incurable ...
Oct 16, 2025
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A novel treatment for Leber's Hereditary Optic Neuropathy (LHON), a rare genetic disease that can lead to sudden loss of vision, has been approved for NHS patients following a successful clinical trial co-led by UCL and Moorfields ...
Sep 15, 2025
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A multinational study led by the LHON Study Group has revealed sustained visual improvements and a favorable safety profile five years following lenadogene nolparvovec gene therapy in patients with Leber hereditary optic ...
In a recent study published in Science China Life Sciences, it has been demonstrated that MSC-mediated mitochondrial transfer can effectively restore mitochondrial DNA (mtDNA) and improve mitochondrial function in neural ...
Aug 29, 2024
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DNA is our body's instruction manual. It contains all the information that our cells need to make proteins and other molecules vital for our development, growth and survival.
Mar 23, 2023
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A 28-patient phase 1 gene therapy clinical trial for the degenerative retinal disease Leber hereditary optic neuropathy (LHON) found no significant safety concerns; however, treatment failed to improve or slow vision loss, ...
Jun 2, 2022
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New research has revealed for the first time the number of Australian families affected by genetic mutations that cause the rare genetic eye disease Leber Hereditary Optic Neuropathy (LHON) – and the risk of going blind from ...
Nov 5, 2021
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Injecting a gene therapy vector into one eye of someone suffering from LHON, the most common cause of mitochondrial blindness, significantly improves vision in both eyes, scientists have found.
Dec 9, 2020
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Leber hereditary optic neuropathy (LHON) is an inherited form of vision loss that causes people to have trouble with their colour vision and difficulty seeing in the centre of their visual field. Due to the founder effect ...
May 24, 2019
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