University of Oxford scientists have developed a gene therapy that has shown promise in treating choroideremia, a rare disorder that causes progressive vision loss, mostly in males.
Doctors tested the treatment method on six patients with choroideremia by injecting numerous healthy genes into the eye to replace missing ones in the retina. The purpose of this therapy is to hinder or prevent loss of sight, according to Oxford’s announcement.
Results varied for each participant. Two patients experienced a big improvement in vision that lasted for four years, but a decline occurred in the other untreated eye. Three separate patients maintained their vision in the treated eye over four years whereas the sixth recipient who was given a lower dose succumbed to vision decline in both eyes.
The study published in The New England Journal of Medicine didn’t mention if any adverse side effects presented themselves in this small trial, but the physicians hope this treatment could work for similar conditions like macular degeneration.
WATCH: A Promising Pipeline for Gene Therapies
The next potential step is testing this treatment out in a larger clinical trial. Popular Science noted this continues a trend of analyzing the efficacy of gene therapies in eye diseases.
One or two genes typically cause vision disorders. Administering the treatments to eyes is not difficult and researchers can easily analyze the results by comparing the treated eye to the untreated eye.
Gene therapy is a burgeoning field. Its potential applications are being tested in curing muscular dystrophy, aging, and even cancer.
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Filed Under: Drug Discovery
