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Two mutations within a feline mucopolysaccharidosis type VI colony cause three different clinical phenotypes.

Mucopolysaccharidosis type VI (MPS VI) is a lysosomal storage disease caused by a deficiency of N-acetylgalactosamine-4-sulfatase (4S). A feline MPS VI model used to demonstrate efficacy of enzyme replacement therapy is due to the homozygous presence of an L476P mutation in 4-sulfatase. An additiona...

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Detalhes bibliográficos
Main Authors: Crawley, A C, Yogalingam, G, Muller, V J, Hopwood, J J
Formato: Artigo
Idioma:Inglês
Publicado em: 1998
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC508546/
https://ncbi.nlm.nih.gov/pubmed/9421472
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