Nonetheless, HITI superexon integration approaches have been used successfully in models of other diseases such as retinitis pigmentosa [21,44] and adrenoleukodystrophy [45], both ABE and HITI have been used to correct Duchenne’s muscular dystrophy in mice [46,47], additional studies of ABE for CFTR splicing mutants have recently been described [48], and clinical testing of ABE has commenced for heterozygous familial hypercholesterolaemia [49]. The gene discussed is CFTR; the disease is retinitis pigmentosa.