Scientists from Trinity College Dublin have developed a highly promising gene therapy to treat glaucoma.
The team has published in the International Journal of Molecular Genetics showing how the gene therapy conferred significant benefit in animal models, and in human cells derived from people with glaucoma.1
Specifically, the therapy protected key retinal ganglion cells (RGCs) and improved their function in an animal model of glaucoma.
The team previously showed its therapy offered promise in treating dry age-related macular degeneration (AMD).
In human retinal cells, delivery of the gene therapy increased oxygen consumption and ATP (energy) production, indicating enhanced cell performance, Trinity College said in a news release.
First author, Dr Sophia MillingtonWard, observed the complexity of treating glaucoma due to its multifactorial condition with many different risk factors and said some patients do not respond to current treatments and/or suffer serious side effects.
“The need for better treatment options has inspired and motivated us to continue developing gene therapies, and we are delighted with the promise it is showing.”
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 and reducing damaging reactive oxygen species.
Based on these and other foundational achievements, Vzarii Therapeutics has been founded to expedite future development of dry AMD and glaucoma gene therapies towards human clinical trials.
Reference available at mivision.com.au.