Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.

Published online

Journal Article

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.

Full Text

Duke Authors

Cited Authors

  • Sun, B; Banugaria, SG; Prater, SN; Patel, TT; Fredrickson, K; Ringler, DJ; de Fougerolles, A; Rosenberg, AS; Waldmann, H; Kishnani, PS

Published Date

  • 2014

Published In

Volume / Issue

  • 1 /

Start / End Page

  • 446 - 450

PubMed ID

  • 27896120

Pubmed Central ID

  • 27896120

International Standard Serial Number (ISSN)

  • 2214-4269

Digital Object Identifier (DOI)

  • 10.1016/j.ymgmr.2014.08.005


  • eng

Conference Location

  • United States