FKRP and autosomal recessive limb-girdle muscular dystrophy type 2I: Using a newly generated immunodeficient FKRP-mutant mouse model, we have shown that transplanted pluripotent stem cell-derived myogenic progenitors are able to engraft, rescue α-DG functional glycosylation, and improve muscle strength, providing proof-of-concept for the potential therapeutic application of stem cell therapy for LGMD2I associated with FKRP mutations.