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Approximately 35-40% of patients with classic infantile Pompe disease treated with enzyme replacement therapy (ERT) develop high, sustained antibody titers against the therapeutic enzyme alglucosidase alfa, which abrogates the treatment efficacy. Induction of antigen-specific immune tolerance would greatly enhance ERT for these patients. Here we show that a short-course treatment with non-depleting anti-CD4 monoclonal antibody successfully induced long-term ERT-specific immune tolerance in Pompe disease mice. Our data suggest an effective adjuvant therapy to ERT.

Original publication




Journal article


Mol Genet Metab Rep

Publication Date





446 - 450


Anti-CD4 antibody, Antigen-specific immune tolerance, CRIM, cross-reacting immunologic material, ERT, enzyme replacement therapy, Enzyme replacement therapy, GAA, acid-α-glucosidase, IPD, infantile Pompe disease, ITI, immune tolerance induction, MTX, methotrexate, Pompe disease