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.

Gene editing is a technology that enables scientists to precisely alter human DNA to treat genetic diseases or to study disease mechanisms in more detail by re-creating the genetic mutation of interest in a cell model.