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Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I.
Wang, Raymond Y; Aminian, Afshin; McEntee, Michael F; Kan, Shih-Hsin; Simonaro, Calogera M; Lamanna, William C; Lawrence, Roger; Ellinwood, N Matthew; Guerra, Catalina; Le, Steven Q; Dickson, Patricia I; Esko, Jeffrey D.
Afiliação
  • Wang RY; Division of Metabolic Disorders, CHOC Children's, Orange, CA, USA. Electronic address: rawang@choc.org.
  • Aminian A; Orthopedic Institute, CHOC Children's, Orange, CA, USA.
  • McEntee MF; Department of Biomedical and Diagnostic Sciences, University of Tennessee, Knoxville, TN, USA.
  • Kan SH; Division of Medical Genetics, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA, USA.
  • Simonaro CM; Departments of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
  • Lamanna WC; Department of Cellular and Molecular Medicine, Glycobiology Research and Training Center, University of California-San Diego, La Jolla, CA, USA.
  • Lawrence R; Department of Cellular and Molecular Medicine, Glycobiology Research and Training Center, University of California-San Diego, La Jolla, CA, USA.
  • Ellinwood NM; Department of Animal Science, Iowa State University, Ames, IA, USA.
  • Guerra C; Biological Resource Center, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA, USA.
  • Le SQ; Division of Medical Genetics, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA, USA.
  • Dickson PI; Division of Medical Genetics, Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, CA, USA.
  • Esko JD; Department of Cellular and Molecular Medicine, Glycobiology Research and Training Center, University of California-San Diego, La Jolla, CA, USA.
Mol Genet Metab ; 112(4): 286-93, 2014 Aug.
Article em En | MEDLINE | ID: mdl-24951454

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Cartilagem Articular / Mucopolissacaridose I / Terapia de Reposição de Enzimas / Glicosaminoglicanos / Iduronidase Tipo de estudo: Prognostic_studies / Qualitative_research Limite: Animals / Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2014 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Cartilagem Articular / Mucopolissacaridose I / Terapia de Reposição de Enzimas / Glicosaminoglicanos / Iduronidase Tipo de estudo: Prognostic_studies / Qualitative_research Limite: Animals / Humans Idioma: En Revista: Mol Genet Metab Assunto da revista: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Ano de publicação: 2014 Tipo de documento: Article