Researchers eye up new gene therapy trial that could reverse hereditary blindness

The two most common inherited optic neuropathies are Leber hereditary optic neuropathy (LHON) and autosomal dominant optic atrophy (DOA).

The management of patients with inherited optic neuropathies remains challenging as there are limited treatment options to slow or prevent ongoing loss of vision.

Dr Yu Wai Man and colleagues have successfully used a gene therapy product to replace the defective gene that causes LHON in patients carrying a specific mutation. Results so far show that those who were treated within 1 year of being affected experienced significant improvement in their vision. These results were published in Science translational medicine. Fortunately therefore, important progress is being made but there are still many questions to address. With his Advanced Fellowship, Patrick will tackle some of the unanswered questions in LHON and DOA.

The trial is funded by an NIHR Moorfields Eye Charity Advanced Fellowship and supported by the NIHR Cambridge Biomedical Research Centre (BRC).

For more information visit: Moorfields Eye Charity Research and Innovation

Published September 1 2021