Gene Therapy Restores Sight in Blind Children with Rare Eye Disorder

Published on Spencer Knight via LinkedIn

A gene therapy trial at Moorfields Eye Hospital in London has given four children born with Leber Congenital Amaurosis (LCA) the ability to see. The therapy used viral vectors to deliver functioning copies of the AIPL1 gene directly into the retina. One of the children, 6-year-old Jace, could previously only detect light, but now he can read, recognize faces, and navigate the world.

This milestone shows gene therapy's expanding reach beyond blood disorders and cancer, offering life-altering results for neurological and sensory conditions too. Spencer Knight calls it “amazing,” and rightly so, restoring vision to blind children is no small feat.

Read the full article on BBC News:

👉 Gene therapy experiment gives children 'life-changing' sight boost

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