Gene therapy works by replacing the defective mutated gene in the affected cells with a normal healthy copy, primarily in inherited retinal disorders.

LCA/EOSRD form part of a spectrum of inherited retinal dystrophies that cause severe visual loss at an early age. There is now an approved retinal gene therapy called Luxturna for patients with RPE65 mutations.

LCA describes a group of inherited retinal conditions that cause severe sight impairment or blindness from birth. There is now an approved treatment called Luxturna for patients with RPE65 mutations.

Treatment centres in the UK currently offering Luxturna treatment under the NHS.