The potential for RNA editing has now been demonstrated in vitro and in vivo for pathogenic mutations in genes related to cystic fibrosis, Duchenne’s muscular dystrophy, Hurler’s syndrome, and Ornithine transcarbamylase (OTC) deficiency, among others [42,47,49,54,74]. The gene discussed is OTC; the disease is ornithine carbamoyltransferase deficiency.