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

Mol Genet Metab. 2002 Sep-Oct;77(1-2):127-35. doi: 10.1016/s1096-7192(02)00148-8.

Abstract

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 alpha-L-iduronidase immunized rats, representing a model of the lysosomal storage disorder Hurler syndrome (alpha-L-iduronidase deficiency). The antibody response to alpha-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 alpha-L-iduronidase was used to reproduce the in vivo effect of altered enzyme tissue distribution, previously observed in immunized rats infused with alpha-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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Antibodies, Monoclonal
  • Disease Models, Animal
  • Epitope Mapping
  • Epitopes / genetics
  • Humans
  • Iduronidase / genetics
  • Iduronidase / immunology*
  • Iduronidase / metabolism
  • Iduronidase / therapeutic use*
  • Immunization
  • Lysosomes / enzymology
  • Mice
  • Molecular Sequence Data
  • Mucopolysaccharidosis I / drug therapy
  • Mucopolysaccharidosis I / enzymology
  • Mucopolysaccharidosis I / immunology
  • Rats
  • Rats, Sprague-Dawley
  • Tissue Distribution

Substances

  • Antibodies, Monoclonal
  • Epitopes
  • Iduronidase