Mutations in ABCA4 are a major cause of STGD, and even though they cover a wide spectrum of severity, most individuals with ABCA4 disease have an intermediate phenotype between the extremes.68 The missense mutation c.5882G>A, p.(G1961E) is the most common mutation of ABCA4 (18.5% in Europe and the United States).69,70 BE or PEs could be used to reverse this mutation to stop or slow down the degeneration of patients' retinas. Here, ABCA4 is linked to severe early-childhood-onset retinal dystrophy.