Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.
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2014
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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.
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Sun, Baodong, Suhrad G Banugaria, Sean N Prater, Trusha T Patel, Keri Fredrickson, Douglas J Ringler, Antonin de Fougerolles, Amy S Rosenberg, et al. (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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Scholars@Duke
Baodong Sun
My overall research interests are finding effective treatment for human inherited metabolic disorders, including glycogen storage diseases (GSDs) and lysosomal storage diseases (LSDs) . My current research focuses on identification of novel therapeutic targets and development of effective therapies for Pompe disease (GSD II) and Cori disease (GSD III) using murine and canine animal disease models. The main therapeutic approaches we are using in our pre-clinical studies include protein/enzyme therapy, AAV-mediated gene therapy, and substrate reduction therapy with small molecule drugs.
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