A Connecticut boy, Jace, is among four children born with severe childhood blindness who have made remarkable improvements in their vision after an experimental trial of gene therapy. The four toddlers were diagnosed with Leber congenital amaurosis (LCA), a rare genetic condition that affects how the retina develops and causes babies to be born blind or with low vision.
Jace was just two months old when his parents, DJ and Brendan, noticed problems with his eyesight. After several tests, he was diagnosed with an aggressive form of LCA at eight weeks old. The family connected with a UK retinal specialist, Michel Michaelides, who led them to the pioneering gene therapy trial.
The trial involved injecting healthy copies of the defective gene into Jace’s eye when he was two years old. The surgery was done in one eye only to minimize potential safety risks. Within a month, Jace showed remarkable improvement in his vision. He could spin, dance, and recognize objects within six months.
Experts are now exploring ways to make the new treatment more widely available. The results of the trial have been reported in the Lancet medical journal, showing dramatic and life-changing improvements in vision for all four toddlers. Dr. Michelides stated that the gene therapy has shown immense potential to change lives and has effectively treated the most severe form of childhood blindness.
Source: https://people.com/toddler-born-blind-gets-vision-restored-after-life-changing-gene-therapy-11684204