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Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency.
Schlotawa, Lars; Tyka, Karolina; Kettwig, Matthias; Ahrens-Nicklas, Rebecca C; Baud, Matthias; Berulava, Tea; Brunetti-Pierri, Nicola; Gagne, Alyssa; Herbst, Zackary M; Maguire, Jean A; Monfregola, Jlenia; Pena, Tonatiuh; Radhakrishnan, Karthikeyan; Schröder, Sophie; Waxman, Elisa A; Ballabio, Andrea; Dierks, Thomas; Fischer, André; French, Deborah L; Gelb, Michael H; Gärtner, Jutta.
Afiliação
  • Schlotawa L; Department of Paediatrics and Adolescent Medicine, University Medical Centre Göttingen, Göttingen, Germany.
  • Tyka K; Department of Paediatrics and Adolescent Medicine, University Medical Centre Göttingen, Göttingen, Germany.
  • Kettwig M; Department of Paediatrics and Adolescent Medicine, University Medical Centre Göttingen, Göttingen, Germany.
  • Ahrens-Nicklas RC; Division of Human Genetics and Metabolism, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Baud M; School of Chemistry and Institute for Life Sciences, University of Southampton, Southampton, UK.
  • Berulava T; Department for Epigenetics and Systems Medicine in Neurodegenerative Diseases, German Centre for Neurodegenerative Diseases, Göttingen, Germany.
  • Brunetti-Pierri N; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Gagne A; Department of Translational Medicine, University of Naples Federico II, Naples, Italy.
  • Herbst ZM; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Maguire JA; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Monfregola J; Department of Chemistry, University of Washington, Seattle, WA, USA.
  • Pena T; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Radhakrishnan K; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Schröder S; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.
  • Waxman EA; Department of Translational Medicine, University of Naples Federico II, Naples, Italy.
  • Ballabio A; Department for Epigenetics and Systems Medicine in Neurodegenerative Diseases, German Centre for Neurodegenerative Diseases, Göttingen, Germany.
  • Dierks T; Bioinformatics Unit, German Centre for Neurodegenerative Diseases, Göttingen, Germany.
  • Fischer A; Faculty of Chemistry, Biochemistry I, Bielefeld University, Bielefeld, Germany.
  • French DL; Department for Epigenetics and Systems Medicine in Neurodegenerative Diseases, German Centre for Neurodegenerative Diseases, Göttingen, Germany.
  • Gelb MH; Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Gärtner J; Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
EMBO Mol Med ; 15(3): e14837, 2023 03 08.
Article em En | MEDLINE | ID: mdl-36789546
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença da Deficiência de Múltiplas Sulfatases Tipo de estudo: Diagnostic_studies / Prognostic_studies / Screening_studies Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença da Deficiência de Múltiplas Sulfatases Tipo de estudo: Diagnostic_studies / Prognostic_studies / Screening_studies Limite: Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article