Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.
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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.
Antigen-specific immune tolerance
CRIM, cross-reacting immunologic material
ERT, enzyme replacement therapy
Enzyme replacement therapy
IPD, infantile Pompe disease
ITI, immune tolerance induction
Published Version (Please cite this version)10.1016/j.ymgmr.2014.08.005
Publication InfoBanugaria, SG; de Fougerolles, A; Fredrickson, Keri B; Kishnani, Priya Sunil; Patel, TT; Prater, SN; ... Waldmann, H (2014). Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease. Mol Genet Metab Rep, 1. pp. 446-450. 10.1016/j.ymgmr.2014.08.005. Retrieved from https://hdl.handle.net/10161/15084.
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Chen Family Professor of Pediatrics
RESEARCH INTERESTS A multidisciplinary approach to care of individuals with genetic disorders in conjunction with clinical and bench research that contributes to: 1) An understanding of the natural history and delineation of long term complications of genetic disorders 2) The development of new therapies for genetic disorders through translational research 3) The development and execution of large multicenter trials to confirm safety and efficacy of potential th
Associate Professor of Pediatrics
My overall research interests are finding effective treatment for human glycogen storage diseases (GSDs) and other inherited metabolic disorders. My current research focuses on identification of novel therapeutic targets and development of effective therapies for GSD II (Pompe disease), GSD III (Cori disease), and GSD IV (Andersen disease) using cellular and animal disease models. The main therapeutic approaches we are using in our pre-clinical studie
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