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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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Autores principales: Sun, Baodong, Banugaria, Suhrad G., Prater, Sean N., Patel, Trusha T., Fredrickson, Keri, Ringler, Douglas J., de Fougerolles, Antonin, Rosenberg, Amy S., Waldmann, Herman, Kishnani, Priya S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5121343/
https://www.ncbi.nlm.nih.gov/pubmed/27896120
http://dx.doi.org/10.1016/j.ymgmr.2014.08.005
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author Sun, Baodong
Banugaria, Suhrad G.
Prater, Sean N.
Patel, Trusha T.
Fredrickson, Keri
Ringler, Douglas J.
de Fougerolles, Antonin
Rosenberg, Amy S.
Waldmann, Herman
Kishnani, Priya S.
author_facet Sun, Baodong
Banugaria, Suhrad G.
Prater, Sean N.
Patel, Trusha T.
Fredrickson, Keri
Ringler, Douglas J.
de Fougerolles, Antonin
Rosenberg, Amy S.
Waldmann, Herman
Kishnani, Priya S.
author_sort Sun, Baodong
collection PubMed
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.
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spelling pubmed-51213432016-11-28 Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease Sun, Baodong Banugaria, Suhrad G. Prater, Sean N. Patel, Trusha T. Fredrickson, Keri Ringler, Douglas J. de Fougerolles, Antonin Rosenberg, Amy S. Waldmann, Herman Kishnani, Priya S. Mol Genet Metab Rep Short Communication 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. Elsevier 2014-10-12 /pmc/articles/PMC5121343/ /pubmed/27896120 http://dx.doi.org/10.1016/j.ymgmr.2014.08.005 Text en © 2014 The Authors http://creativecommons.org/licenses/by-nc-nd/3.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/3.0/).
spellingShingle Short Communication
Sun, Baodong
Banugaria, Suhrad G.
Prater, Sean N.
Patel, Trusha T.
Fredrickson, Keri
Ringler, Douglas J.
de Fougerolles, Antonin
Rosenberg, Amy S.
Waldmann, Herman
Kishnani, Priya S.
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease
title 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_short 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
topic Short Communication
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5121343/
https://www.ncbi.nlm.nih.gov/pubmed/27896120
http://dx.doi.org/10.1016/j.ymgmr.2014.08.005
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