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1,6-epi-Cyclophellitol Cyclosulfamidate Is a Bona Fide Lysosomal α-Glucosidase Stabilizer for the Treatment of Pompe Disease.
Kok, Ken; Kuo, Chi-Lin; Katzy, Rebecca E; Lelieveld, Lindsey T; Wu, Liang; Roig-Zamboni, Véronique; van der Marel, Gijsbert A; Codée, Jeroen D C; Sulzenbacher, Gerlind; Davies, Gideon J; Overkleeft, Herman S; Aerts, Johannes M F G; Artola, Marta.
Afiliação
  • Kok K; Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Kuo CL; Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Katzy RE; Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Lelieveld LT; Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Wu L; Department of Chemistry, University of York, York YO10 5DD, U.K.
  • Roig-Zamboni V; Architecture et Fonction des Macromolécules Biologiques (AFMB), CNRS, Aix-Marseille University, Marseille 13288, France.
  • van der Marel GA; Department of Bio-Organic Synthesis, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Codée JDC; Department of Bio-Organic Synthesis, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Sulzenbacher G; Architecture et Fonction des Macromolécules Biologiques (AFMB), CNRS, Aix-Marseille University, Marseille 13288, France.
  • Davies GJ; Department of Chemistry, University of York, York YO10 5DD, U.K.
  • Overkleeft HS; Department of Bio-Organic Synthesis, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Aerts JMFG; Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
  • Artola M; Department of Medical Biochemistry, Leiden Institute of Chemistry, Leiden University, Einsteinweg 55, Leiden 2333 CC, The Netherlands.
J Am Chem Soc ; 144(32): 14819-14827, 2022 08 17.
Article em En | MEDLINE | ID: mdl-35917590
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article