New gene therapy offers promise for treating glaucoma 

Key Takeaways

• Scientists have developed a highly promising gene therapy to treat glaucoma.
• The therapy conferred significant benefit in both animal models and human cells derived from people with glaucoma.

Scientists from Trinity College Dublin have developed a highly promising gene therapy to treat glaucoma. Their work, published in the International Journal of Molecular Genetics, shows that the gene therapy conferred significant benefit in animal models, and in human cells derived from people with glaucoma. Specifically, the therapy protected key “retinal ganglion cells” (RGCs) that are important in vision and improved their function in an animal model of glaucoma. In human retinal cells, delivery of the gene therapy increased oxygen consumption and energy production, indicating enhanced cell performance.

The new gene therapy uses an approved virus to deliver an enhanced gene (eNdi1) developed by the Trinity team. The therapy was designed with the aim of boosting mitochondrial activity (mitochondria are “cellular energy generators”) and reducing damaging reactive oxygen species. 

Translation of the studies towards the clinic and patients, while involving many additional steps, is the next focus. Based on these and other foundational achievements, the Trinity team recently founded Vzarii Therapeutics to expedite future development of the glaucoma gene therapy towards human clinical trials.

Edited by Miriam Kaplan, PhD

Source:

Trinity College Dublin, ScienceDaily, September 9, 2024; see source article