Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease
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...
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2014-01-01
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| Series: | Molecular Genetics and Metabolism Reports |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2214426914000561 |
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doaj-e2218e4187334a8baee1035af47e2f372020-11-24T22:28:52ZengElsevierMolecular Genetics and Metabolism Reports2214-42692014-01-011C44645010.1016/j.ymgmr.2014.08.005Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe diseaseBaodong Sun0Suhrad G. Banugaria1Sean N. Prater2Trusha T. Patel3Keri Fredrickson4Douglas J. Ringler5Antonin de Fougerolles6Amy S. Rosenberg7Herman Waldmann8Priya S. Kishnani9Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USADivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USADivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USADivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USADivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USATolerx, Inc., Cambridge, MA, USATolerx, Inc., Cambridge, MA, USADivision of Therapeutic Proteins, U.S. Food and Drug Administration, Silver Spring, MD, USASir William Dunn School of Pathology, Oxford University, Oxford, UKDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USAApproximately 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.http://www.sciencedirect.com/science/article/pii/S2214426914000561Pompe diseaseAnti-CD4 antibodyAntigen-specific immune toleranceEnzyme replacement therapy |
| collection |
DOAJ |
| language |
English |
| format |
Article |
| sources |
DOAJ |
| author |
Baodong Sun Suhrad G. Banugaria Sean N. Prater Trusha T. Patel Keri Fredrickson Douglas J. Ringler Antonin de Fougerolles Amy S. Rosenberg Herman Waldmann Priya S. Kishnani |
| spellingShingle |
Baodong Sun Suhrad G. Banugaria Sean N. Prater Trusha T. Patel Keri Fredrickson Douglas J. Ringler Antonin de Fougerolles Amy S. Rosenberg Herman Waldmann Priya S. Kishnani Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease Molecular Genetics and Metabolism Reports Pompe disease Anti-CD4 antibody Antigen-specific immune tolerance Enzyme replacement therapy |
| author_facet |
Baodong Sun Suhrad G. Banugaria Sean N. Prater Trusha T. Patel Keri Fredrickson Douglas J. Ringler Antonin de Fougerolles Amy S. Rosenberg Herman Waldmann Priya S. Kishnani |
| author_sort |
Baodong Sun |
| title |
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease |
| title_short |
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease |
| title_full |
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease |
| title_fullStr |
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease |
| title_full_unstemmed |
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease |
| title_sort |
non-depleting anti-cd4 monoclonal antibody induces immune tolerance to ert in a murine model of pompe disease |
| publisher |
Elsevier |
| series |
Molecular Genetics and Metabolism Reports |
| issn |
2214-4269 |
| publishDate |
2014-01-01 |
| description |
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. |
| topic |
Pompe disease Anti-CD4 antibody Antigen-specific immune tolerance Enzyme replacement therapy |
| url |
http://www.sciencedirect.com/science/article/pii/S2214426914000561 |
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