An experimental trial of gene therapy has helped four toddlers born with severe forms of childhood blindness gain significant improvements in their sight. The rare genetic condition causes rapid vision deterioration from birth, leaving babies registered as legally blind and only able to distinguish between dark and light.
The innovative procedure involves injecting healthy copies of a defective gene into the back of a child’s eye early in life to treat a severe form of Leber Congenital Amaurosis. Gene therapy has been available on the NHS since 2020 for another genetic blindness condition.
In London, four children from the US, Turkey, and Tunisia underwent the treatment at Moorfields Eye Hospital under a special licence designed for compassionate use. The procedure was quick and easy, with tiny scars in their eye where healthy copies of the gene were injected through keyhole surgery.
After just one month following treatment, parents reported significant improvements in their children’s sight, including Jace, from Connecticut who could previously only track objects at all. His progress has been “amazing,” with his parents noticing him squinting for the first time upon seeing bright sunshine and picking up toys off the floor.
The results of the trial give hope that intervening early in other childhood genetic eye conditions could offer significant benefits. Doctors believe treatment at this stage can transform children’s lives, allowing them to interact more effectively with people and develop normally. Further work is planned to confirm the findings and monitor the long-term effects of the gene therapy.
Source: https://www.bbc.com/news/articles/c5ydnz2d75xo