| First Author | Vogler C | Year | 1993 |
| Journal | Pediatr Res | Volume | 34 |
| Issue | 6 | Pages | 837-40 |
| PubMed ID | 8108204 | Mgi Jnum | J:22453 |
| Mgi Id | MGI:66556 | Doi | 10.1203/00006450-199312000-00028 |
| Citation | Vogler C, et al. (1993) Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse. Pediatr Res 34(6):837-40 |
| abstractText | 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. |
