Immune response to enzyme replacement therapy: Single epitope control of antigen distribution from circulation

Elias N. Glaros, Chris T. Turner, Emma J. Parkinson, John J. Hopwood, Doug A. Brooks

Research output: Contribution to journalArticlepeer-review

8 Citations (Scopus)


Immune response to replacement therapy has been reported for a range of therapeutic strategies being developed for the treatment of patients with genetic disease. The potential problem of immune response to enzyme replacement therapy has been investigated in α-L-iduronidase immunized rats, representing a model of the lysosomal storage disorder Hurler syndrome (α-Liduronidase deficiency). The antibody response to α-L-iduronidase showed that the positional location of antibody reactivity was similar for different immunized rats, but the precise linear sequence epitopes identified, varied between rats. A monoclonal antibody reacting to an epitope in close proximity to one high antigenicity site on α-L-iduronidase was used to reproduce the in vivo effect of altered enzyme tissue distribution, previously observed in immunized rats infused with α-L-iduronidase. The study demonstrated that during an immune response, antibody reacting to a single epitope could partially control the tissue distribution of antigen from circulation.

Original languageEnglish
Pages (from-to)127-135
Number of pages9
JournalMolecular Genetics and Metabolism
Issue number1-2
Publication statusPublished or Issued - 2002


  • Antibodies
  • Epitopes
  • Genetic disease
  • Immune response
  • Therapy

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism
  • Biochemistry
  • Molecular Biology
  • Genetics
  • Endocrinology

Cite this