FKRP evaluation by WB analysis highlighted an increasing trend of protein expression between MOI of 10 and 20, while the MOI of 40 seemed to represent a steady point leading to FKRP expression plateau ( Fig. 2M ).
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Autologous intramuscular transplantation of engineered satellite cells induces exosome-mediated systemic expression of Fukutin-related protein and rescues disease phenotype in a murine model of limb-girdle muscular dystrophy type 2I.
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