To investigate whether other mutations associated with the WWS phenotype are amenable to rescue by these metabolites, we introduced the WWS-clinically associated FKRP-C318Y mutation (Beltran-Valero de Bernabé et al., 2004) located in the zinc finger loop of the FKRP catalytic domain into WT iPS cells using CRISPR-Cas9 genome editing. Here, FKRP is linked to muscular dystrophy-dystroglycanopathy, type A.