Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse

Pediatr Res. 1993 Dec;34(6):837-40. doi: 10.1203/00006450-199312000-00028.

Abstract

beta-Glucuronidase injected i.v. into newborn mucopolysaccharidosis VII mice was cleared from the circulation in less than 1 h and taken up by tissues in a distribution corresponding to the location of the mannose 6-phosphate receptor. One h after a 3.5-mg/kg beta-glucuronidase injection, beta-glucuronidase levels were equal to or greater than normal in every organ examined with the exception of the brain, where 31% normal activity was present. Enzyme was detectable histochemically in the major sites of pathology for mucopolysaccharidosis VII including bone, brain, heart, and fixed tissue macrophages. The half-life of recombinant beta-glucuronidase activity in various organs of injected mucopolysaccharidosis VII mice was 1.5 to 4.5 d. These studies show that recombinant beta-glucuronidase administered to newborn mice reaches the sites of clinically important storage in murine mucopolysaccharidosis VII.

Publication types

  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Animals
  • Animals, Newborn
  • Glucuronidase / administration & dosage
  • Glucuronidase / pharmacokinetics
  • Glucuronidase / therapeutic use*
  • Half-Life
  • Histocytochemistry
  • Injections, Intravenous
  • Mice
  • Mice, Mutant Strains
  • Mucopolysaccharidosis VII / drug therapy*
  • Mucopolysaccharidosis VII / enzymology
  • Mucopolysaccharidosis VII / genetics
  • Recombinant Proteins / administration & dosage
  • Recombinant Proteins / pharmacokinetics
  • Recombinant Proteins / therapeutic use
  • Tissue Distribution

Substances

  • Recombinant Proteins
  • Glucuronidase