Researchers from the University College London (UCL) used gene therapy to partly restore the function of the retina’s cone receptors in two children, according to a press release published by the institution.
This is a promising step toward reactivating previously inactive communication pathways between the retina and the brain by utilizing the plasticity of the teenage brain.
The research employs a novel method to determine if the medication alters the brain circuits particular to the cones in children with achromatopsia, a partial or total absence of color vision.
“We are still analyzing the results from our two clinical trials to see whether this gene therapy can effectively improve everyday vision for people with achromatopsia. We hope that with positive results, and with further clinical trials, we could greatly improve the sight of people with inherited retinal diseases,” said Dr. Michel Michaelides, the co-lead author of the study.
Related on the SLP
The results of the study were published in the journal Brain.
Abstract:
Recent advances in regenerative therapy have placed the treatment of previously incurable eye diseases within arms’ reach. Achromatopsia is a severe monogenic heritable retinal disease that disrupts cone function from birth, leaving patients with complete color blindness, low acuity, photosensitivity and nystagmus.
