Gene Therapy Shows Potential for Repairing Damage Caused by Glaucoma and Dementia

A study in Science Advances demonstrated how gene therapy might help correct damage caused by dementia and glaucoma, reports Genetic Engineering & Biotechnology News . Earlier research found brain-derived neurotrophic factor (BDNF) and its receptor tropomyosin receptor kinase B (TrkB) held promise for enhancing axonal function in damaged nerve cells. The researchers determined that axonal transport between the eye and brain was impaired in an animal model of glaucoma, while a decline in electrical activity in the retina in response to light suggested that vision was also affected. The team used a viral vector system to deliver TrkB and BDNF to the retina of glaucoma model rats, which restored axonal transport between the retina and the brain and improved electrical response to light. The same therapy also restored axonal transport between the eye and brain in mice engineered to model the accumulation of tau protein tangles in the brain, indicating possible improvement in short-term memory. "Given the recent correlation between Alzheimer's pathology in the brain and retina, this approach shows promise as an overall strategy to target intrinsic neuronal mechanisms to improve neuronal repair while overcoming several problems relating to ligand deficiency and receptor down-regulation and dysfunction that may be relevant to multiple neurodegenerative diseases," the team concluded.