The treatment of many diseases has been improved by the use of genetic technology in medicine. Leber Congenital Amaurosis (LCA) is an inherited eye disease. In LCA, the photoreceptor cells in the retina die at an early age. This causes impaired vision (reduced eyesight) in children, which can progress to blindness. Mutations in different genes cause different forms of LCA. One form of this disease, LCA2, is caused by a mutation in the RPE65 gene. Gene therapy has been used to treat LCA2. Outline how an inherited eye disease, such as LCA2, is treated with gene therapy. ....................................................................................................................................... . ....................................................................................................................................... . ....................................................................................................................................... . ....................................................................................................................................... . ....................................................................................................................................... . ....................................................................................................................................... . ....................................................................................................................................... . ................................................................................................................................. (ii) Suggest why the eye is a suitable organ for gene therapy. ....................................................................................................................................... . .................................................................................................................................

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