Abstract | INTRODUCTION: RESULTS: All intrathecal injections (IT-INJs) were well tolerated. MPS-VI cats treated with IT-INJ of recombinant human N-acetylgalactosamine-4-sulfatase (rhASB) exhibited reduced vacuolation in the dural fibroblasts, diminished levels of sulfated-N-acetylhexosamine (HNAc(+S)) in the cerebrospinal fluid (CSF) and no hind-limb paresis. Serum anti-rhASB antibodies remained low in MPS-VI cats treated with intravenous enzyme replacement therapy (IV-ERT) and increased slightly in normal cats treated with IT-INJ of rhASB alone. Anti-rhASB antibodies in CSF remained undetectable. DISCUSSION: These data indicate that repeated IT-INJ of rhASB can safely prevent GAG storage in MPS-VI dura. METHODS: Cats were assigned to three groups: (i) receiving weekly IV-ERT of rhASB from birth plus six monthly IT-INJs of rhASB from age 2 months; (ii) receiving six monthly IT-INJs of vehicle; or (iii) untreated. Additional normal cats received five fortnightly IT-INJs of rhASB or vehicle alone.
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Authors | Dyane Auclair, John Finnie, Steven U Walkley, Joleen White, Timothy Nielsen, Maria Fuller, Alphonsus Cheng, Charles A O'Neill, John J Hopwood |
Journal | Pediatric research
(Pediatr Res)
Vol. 71
Issue 1
Pg. 39-45
(Jan 2012)
ISSN: 1530-0447 [Electronic] United States |
PMID | 22289849
(Publication Type: Journal Article)
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Chemical References |
- Glycosaminoglycans
- Recombinant Proteins
- N-Acetylgalactosamine-4-Sulfatase
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Topics |
- Animals
- Cats
- Disease Models, Animal
- Dura Mater
(metabolism, pathology)
- Glycosaminoglycans
(metabolism)
- Humans
- Injections, Spinal
- Mucopolysaccharidosis VI
(drug therapy, enzymology, pathology, physiopathology)
- N-Acetylgalactosamine-4-Sulfatase
(administration & dosage, genetics, therapeutic use)
- Recombinant Proteins
(administration & dosage, therapeutic use)
- Treatment Outcome
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