Leber Hereditary Optic Neuropathy News and Research

RSS

Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdles

Recombinant adeno-associated virus (rAAV)-based genetic applications to treat human diseases.

5 Apr 2024

Gene therapy for degenerative retinal disease has no safety concerns but fails to improve vision loss

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.

1 Jun 2022

Researchers develop new platform capable of mitochondrial DNA editing

Researchers from the Center for Genome Engineering within the Institute for Basic Science developed a new gene-editing platform called transcription activator-like effector-linked deaminases, or TALED.

25 Apr 2022

UC San Diego researchers launch clinical trial to test gene therapy for Alzheimer’s disease

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimer's disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

18 Feb 2021

New data shows encouraging results for patients with neuro-degenerative blinding eye disease

New data published in the Journal Science Translational Medicine shows encouraging results in a worldwide clinical trial for patients diagnosed with the neuro-degenerative blinding eye disease; Leber Hereditary Optic Neuropathy (LHON.)

16 Dec 2020

Study offers important step to identify cause of blinding disease

Leber hereditary optic neuropathy is an inherited form of vision loss that causes people to have trouble with their color vision and difficulty seeing in the center of their visual field.

24 May 2019

Fight for Sight awards new grants for 13 vital eye research projects

Fight for Sight, the leading eye research charity, has awarded grants totaling over £180,000 for thirteen vital research projects in partnership with nine different organizations.

2 Feb 2018

Variations in ancient mitochondrial DNA may play key role in predisposition to ASD

Mitochondria, the tiny structures inside our cells that generate energy, may play a key role in autism spectrum disorders (ASD).

23 Aug 2017

NIH-funded researchers test new gene therapy to improve visual function in mice

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.

6 Oct 2015

Study opens door to possible treatments for variety of mitochondrial diseases

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. The research, published online today in Nature Medicine, was funded by the National Eye Institute, a part of the National Institutes of Health.

5 Aug 2013

Novel technological treatment for Leber Hereditary Optic Neuropathy

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.

24 Apr 2012