Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome
| Autor: | John J. Hopwood, Don S. Anson, Doug A. Brooks, Birgit A. Petersen, Elizabeth L. Isaac, Allison C. Crawley, Alison J. Moore, Nick L. Fazzalari, Barbara King, Vivienne Muller, Julie Bielicki, Sharon Byers, Christine D. Boulter |
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| Přispěvatelé: | Crawley, Allison C, Muller, Vivienne J, Petersen, Birgit A, Isaac, Elizabeth L, Bielicki, Julie, King, Barbara M, Boulter, Christine D, Moore, Alison J, Fazzalari, Nick L, Anson, Donald S, Byers, Sharon, Hopwood, John J |
| Rok vydání: | 1996 |
| Předmět: |
Cartilage
Articular medicine.medical_specialty Time Factors Kupffer Cells Metabolic Clearance Rate Mucopolysaccharidosis type VI CHO Cells Kidney Transfection Chondro-4-Sulfatase Cricetinae Internal medicine Animals Humans Medicine Tissue Distribution Infusions Intravenous Glycosaminoglycans Mucopolysaccharidosis VI CATS business.industry Cartilage General Medicine Enzyme replacement therapy medicine.disease Recombinant Proteins N-Acetylgalactosamine-4-Sulfatase Maroteaux–Lamy syndrome Disease Models Animal Microscopy Electron Endocrinology medicine.anatomical_structure Liver Cats Lysosomes business Half-Life Research Article |
| Zdroj: | Journal of Clinical Investigation. 97:1864-1873 |
| ISSN: | 0021-9738 |
| DOI: | 10.1172/jci118617 |
| Popis: | We report studies that suggest enzyme replacement therapy will result in a significant reduction in disease progression and tissue pathology in patients with Maroteaux-Lamy syndrome (Mucopolysaccharidosis type VI, MPS VI). A feline model for MPS VI was used to evaluate tissue distribution and clinical efficacy of three forms of recombinant human N-acetylgalactosamine-4-sulfatase (rh4S, EC 3.1.6.1). Intravenously administered rh4S was rapidly cleared from circulation. The majority of rh4S was distributed to liver, but was also detected in most other tissues. Tissue half-life was approximately 2-4 d. Three MPS VI cats given regular intravenous infusions of rh4S for up to 20 mo showed variable reduction of storage vacuoles in Kupffer cells and connective tissues, however cartilage chondrocytes remained vacuolated. Vertebral bone mineral volume was improved in two MPS VI cats in which therapy was initiated before skeletal maturity, and increased bone volume appeared to correlate with earlier age of onset of therapy. One cat showed greater mobility in response to therapy. |
| Databáze: | OpenAIRE |
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