Unlocking Sight: How Gene Therapy is Revolutionizing Glaucoma Treatment!
gene therapy glaucoma vision restoration
New Gene Therapy for Glaucoma Could Help Restore Vision
Researchers at Trinity College Dublin have unveiled a novel gene therapy for glaucoma, a breakthrough that safeguards retinal cells and enhances mitochondrial function, potentially revolutionizing current treatment methods.
This pioneering therapy targets glaucoma, a debilitating eye disease that affects nearly 80 million individuals worldwide and can lead to irreversible blindness. Previously, the research team demonstrated the efficacy of this approach in treating dry age-related macular degeneration (AMD).
The findings, now published in the International Journal of Molecular Genetics, reveal that the therapy offers significant advantages in animal models and in human cells derived from glaucoma patients.
Specifically, the gene therapy demonstrated protection for “retinal ganglion cells” (RGCs) crucial for vision, enhancing their function in an animal glaucoma model. Furthermore, in human retinal cells, the gene therapy boosted oxygen consumption and ATP production, suggesting improved cellular performance.
gene therapy glaucoma vision restoration: Urgent Need for Innovative Glaucoma Treatments
Dr. Sophia Millington-Ward, a Research Fellow in Trinity’s School of Genetics and Microbiology and the study’s lead author, emphasizes the urgency for new glaucoma treatments: “Glaucoma, a complex group of optic neuropathies, remains a leading cause of blindness. In Europe, about 1 in 30 individuals aged 40 to 80, and 1 in 10 over 90, suffer from this common condition, underscoring the dire need for alternative treatment options.”
She further explains, “It is a multifactorial disease influenced by numerous risk factors, complicating treatment efforts. Current strategies, including topical eye drops, surgery, and laser therapy, yield inconsistent outcomes, with many patients either not responding or experiencing severe side effects.”
“Our motivation to pursue gene therapy arises from this critical need for more effective treatments, and we are thrilled by the potential it shows,” she adds.
gene therapy glaucoma vision restoration: The Potential of Gene Therapy
This innovative therapy employs a sanctioned virus to deliver an enhanced gene (eNdi1) engineered by the Trinity team, aiming to amplify mitochondrial function—mitochondria being the “powerhouses” of cells that produce ATP—and mitigate harmful reactive oxygen species.
Jane Farrar, Research Professor at Trinity’s School of Genetics and Microbiology and senior author of the study, notes, “Developing versatile gene therapies that can serve a broad patient base is particularly vital given the substantial costs of creating each therapy. Our work suggests that this therapy holds genuine promise in improving mitochondrial function for glaucoma patients.”
The next phase involves translating these findings into clinical applications, a process requiring further steps and refinements.
Building on these foundational successes, the Trinity team, in collaboration with Loretto Callaghan, has established Vzarii Therapeutics to accelerate the development of gene therapies for dry AMD and glaucoma towards clinical trials.
Reference:
“AAV-NDI1 Therapy Provides Significant Benefit to Murine and Cellular Models of Glaucoma” by Sophia Millington-Ward et al., published August 14, 2024, in the International Journal of Molecular Sciences. DOI: 10.3390/ijms25168876.
Funding
This research was supported by Science Foundation Ireland, Enterprise Ireland, Health Research Board Ireland, EU Marie Curie Innovative Training Network, and Fighting Blindness Ireland—Health Research Charities Ireland.
