Glaucoma May Be Treatable by Gene Therapy


A new study led by the University of Bristol in the United Kingdom has shown that glaucoma could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness, and quality of life for many patients. The research team, led by academics at the Bristol Medical School: Translational Health Sciences, tested a new approach that could provide additional treatment options and benefits. Their findings were published in the journal Molecular Therapy.

The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue. The treatment targeted the ciliary body, which produces the fluid that maintains pressure within the eye. Using the gene editing technology CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated, leading to reduced eye pressure.

Dr. Colin Chu, visiting senior research fellow in the Bristol Medical School: Translational Health Sciences and corresponding author, said, “Currently there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early. We hope to advance towards clinical trials for this new treatment in the near future. If it’s successful, it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money.”