F7 and hyperinsulinemic hypoglycemia, familial, 4: Similar approaches have been reported for ATP8B1 deficiency (van der Woerd et al., 2015), FD (Donadon et al., 2018b), Sanfilippo syndrome type C (Matos et al., 2014), Fanconi anemia (Mattioli et al., 2014), RP (Tanner et al., 2009), thalassemia (Gorman et al., 2000), severe coagulation factor VII (FVII) deficiency (hemophilia A, HA) (Pinotti et al., 2008; Donadon et al., 2018a; Balestra et al., 2019b), CDKL5-deficiency disorder (CDD) (Balestra et al., 2019a), Seckel syndrome (Scalet et al., 2017), and hereditary tyrosinemia type I (HT1) (Scalet et al., 2018).