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.

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.

RNA is involved in protein production from DNA in human cells. Mutations in DNA can be passed down to RNA, and a non-functional protein might be produced. RNA therapies target these "mistakes" in the RNA so that a normal functional protein can be produced instead.