Faculty Research 1990 - 1999


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

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Animals-Newborn, Glucuronidase, Half-Life, Histocytochemistry, Injections-Intravenous, Mice, Mice-Mutant-Strains, Mucopolysaccharidosis-VII: dt, en, ge, Recombinant-Proteins, SUPPORT-U-S-GOVT-P-H-S, Tissue-Distribution

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Pediatr Res 1993 Dec;34(6):837-40


DK41082/DK/NIDDK, DK40163/DK/NIDDK, GM34182/GM/NIGMS, +


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.

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