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Type: Journal article
Title: Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome
Author: Crawley, A.
Brooks, D.
Muller, V.
Petersen, B.
Isaac, E.
Bielicki, J.
King, B.
Boulter, C.
Moore, A.
Fazzalari, N.
Anson, D.
Byers, S.
Hopwood, J.
Citation: Journal of Clinical Investigation, 1996; 97(8):1864-1873
Issue Date: 1996
ISSN: 0021-9738
Statement of
Allison C. Crawley, Doug A. Brooks, Vivienne J. Muller, Birgit A. Petersen, Elizabeth L. Isaac, Julie Bielicki, Barbara M. King, Christine D. Boulter, Alison J. Moore, Nick L. Fazzalari, Don S. Anson, Sharon Byers, and John J. Hopwood
Abstract: 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 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.
Keywords: Cartilage, Articular; Liver; Kidney; CHO Cells; Lysosomes; Kupffer Cells; Animals; Cats; Humans; Mucopolysaccharidosis VI; Disease Models, Animal; Chondro-4-Sulfatase; Glycosaminoglycans; Recombinant Proteins; Microscopy, Electron; Metabolic Clearance Rate; Infusions, Intravenous; Transfection; Tissue Distribution; Half-Life; Time Factors; Cricetinae
RMID: 0030005544
DOI: 10.1172/JCI118617
Appears in Collections:Paediatrics publications

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