‘Life-Changing’ Gene Therapy Restores Sight in Children Born Blind
An experimental gene therapy has helped four toddlers regain significant vision improvements after being born with a rare and severe form of childhood blindness, according to doctors at Moorfields Eye Hospital in London. The children, from the U.S., Turkey, and Tunisia, were diagnosed with an aggressive form of Leber Congenital Amaurosis, caused by a mutation in the AIPL1 gene. Before the therapy, they were legally blind, unable to distinguish light and dark. After receiving the treatment, their vision improved, with some now able to draw and write.
The therapy involves injecting healthy copies of the faulty gene into the retina via keyhole surgery, allowing retinal cells to function better and survive longer. Jace, a six-year-old from Connecticut, underwent the procedure at age two and has since shown remarkable progress, now able to track objects and interact visually with the world around him.
Experts say early intervention could transform children’s lives, significantly aiding their development and interaction. The study, published in The Lancet, provides compelling evidence of the treatment’s effectiveness, with the untreated eyes of the children continuing to deteriorate as expected. Doctors plan to monitor the children for long-term effects, hoping that early genetic intervention can help treat other childhood blindness conditions in the future.
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