https://medicalxpress.com/ en-us Medical Xpress internet news portal provides the latest news on Health and Medicine. 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 Veterinary Medicine and Edgene Co., Ltd. https://medicalxpress.com/news/2026-02-gene-therapy-effective-leber-hereditary.html Wed, 04 Feb 2026 22:10:01 EST news689436330 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 mitochondrial diseases. https://medicalxpress.com/news/2025-10-reveals-possibility-drug-therapy-hereditary.html Thu, 16 Oct 2025 15:20:01 EDT news679846599 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 researchers. https://medicalxpress.com/news/2025-09-treatment-rare-genetic-sudden-vision.html Mon, 15 Sep 2025 15:10:08 EDT news677167560 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 neuropathy (LHON) caused by the MT-ND4 gene mutation. https://medicalxpress.com/news/2024-12-sustained-visual-lhon-patients-aav.html Fri, 27 Dec 2024 07:00:01 EST news654497701 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 progenitor cells derived from patients with Leber's hereditary optic neuropathy (LHON). https://medicalxpress.com/news/2024-08-msc-mitochondrial-therapeutic-leber-hereditary.html Thu, 29 Aug 2024 17:06:03 EDT news644169962 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. https://medicalxpress.com/news/2023-03-misplaced-dna-disease.html Thu, 23 Mar 2023 09:45:04 EDT news598783501 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, with even the highest dose. LHON affects the optic nerve, which carries visual signals from the light-sensing retina to the brain. The study, published in the American Journal of Ophthalmology, was sponsored in part by the National Eye Institute, a part of the National Institutes of Health. https://medicalxpress.com/news/2022-06-gene-therapy-rare-eye-disease.html Thu, 02 Jun 2022 10:26:30 EDT news573384388 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 the disease. https://medicalxpress.com/news/2021-11-australia-snapshot-families-rare-genetic.html Fri, 05 Nov 2021 08:46:51 EDT news555320804 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. https://medicalxpress.com/news/2020-12-gene-therapy-eye-scientists-vision.html Wed, 09 Dec 2020 14:00:05 EST news526721252 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 from the filles du roi, there is a disproportionate preponderance of a particular LHON mutation among the French-Canadian population. https://medicalxpress.com/news/2019-05-closer-disease.html Fri, 24 May 2019 02:06:14 EDT news477882367 There have been a lot of theories about what causes Alzheimer's disease. Many of them have given rise to experimental treatments of one form or another. None of them have worked much better than taking anything you might find in your spice rack. https://medicalxpress.com/news/2018-05-alzheimer.html Wed, 23 May 2018 09:10:02 EDT news446279787 Mitochondria, the tiny structures inside our cells that generate energy, may play a key role in autism spectrum disorders (ASD). A provocative new study by Children's Hospital of Philadelphia (CHOP)'s pioneering mitochondrial medicine team suggests that variations in mitochondrial DNA (mtDNA) originating during ancient human migrations may play an important role in predisposition to ASDs. https://medicalxpress.com/news/2017-08-mitochondria-autism.html Wed, 23 Aug 2017 11:10:28 EDT news422704390 Researchers funded by the National Institutes of Health have developed a novel mouse model for the vision disorder Leber hereditary optic neuropathy (LHON), and found that they can use gene therapy to improve visual function in the mice. LHON is one of many diseases tied to gene mutations that damage the tiny energy factories that power our cells, called mitochondria. https://medicalxpress.com/news/2015-10-scientists-gene-therapy-vision-loss.html Mon, 05 Oct 2015 15:00:02 EDT news363264141 (Medical Xpress)—Programmers typically evolve new code by copying and modifying existing code to meet new needs. With the more advanced programming languages, they also make use of something known in the business as polymorphism—the ability to process objects differently depending on their data type or class. Similarly, one way that life evolves is to copy and modify genes. Biologists, however, often use the term polymorphism to mean different things. Sometimes it simply means a non disease-causing change to a base pair, and sometimes it more specifically means a change found at a frequency of 1% or higher in the population. https://medicalxpress.com/news/2015-01-mitochondrial-dna-mutations-good-bad.html Tue, 13 Jan 2015 13:00:02 EST news340374692 By borrowing a tool from bacteria that infect plants, scientists have developed a new approach to eliminate mutated DNA inside mitochondria—the energy factories within cells. Doctors might someday use the approach to treat a variety of mitochondrial diseases, including the degenerative eye disease Leber hereditary optic neuropathy (LHON). The research, published online today in Nature Medicine, was funded by the National Eye Institute (NEI), a part of the National Institutes of Health (NIH). https://medicalxpress.com/news/2013-08-mechanism-bacteria-infect-eye-disease.html Sun, 04 Aug 2013 13:00:23 EDT news294761266 A research team, led by John Guy, M.D., professor of ophthalmology at Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, has pioneered a novel technological treatment for Leber Hereditary Optic Neuropathy (LHON), an inherited genetic defect that causes rapid, permanent, and bilateral loss of vision in people of all ages, but primarily males ages 20-40. Genetic mutations in the mitochondria (part of the cell that produces energy) cause the disorder. Currently, there is no cure for LHON. https://medicalxpress.com/news/2012-04-team-breakthrough-degenerative-vision-disorder.html Mon, 23 Apr 2012 17:06:00 EDT news254419552 A clinical trial led by Newcastle University shows that the drug, idebenone (Catena), improved the vision and perception of colour in patients with Leber's Hereditary Optic Neuropathy (LHON). The inherited condition means patients, who can see normally, lose the sight in one eye then within 3 to 6 months lose the sight in their other eye. https://medicalxpress.com/news/2011-07-drug-shown-sight-patients-inherited.html Tue, 26 Jul 2011 03:35:46 EDT news230869417