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Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.
Li, Yedda; Xu, Yue; Benitez, Bruno A; Nagree, Murtaza S; Dearborn, Joshua T; Jiang, Xuntian; Guzman, Miguel A; Woloszynek, Josh C; Giaramita, Alex; Yip, Bryan K; Elsbernd, Joseph; Babcock, Michael C; Lo, Melanie; Fowler, Stephen C; Wozniak, David F; Vogler, Carole A; Medin, Jeffrey A; Crawford, Brett E; Sands, Mark S.
Afiliación
  • Li Y; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.
  • Xu Y; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
  • Benitez BA; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.
  • Nagree MS; Department of Medical Biophysics, University of Toronto, Toronto, ON M5S, Canada.
  • Dearborn JT; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.
  • Jiang X; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.
  • Guzman MA; Department of Pathology, St. Louis University School of Medicine, St. Louis, MO 63104.
  • Woloszynek JC; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
  • Giaramita A; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
  • Yip BK; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
  • Elsbernd J; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
  • Babcock MC; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
  • Lo M; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
  • Fowler SC; Department of Pharmacology and Toxicology, University of Kansas, Lawrence, KS 66045.
  • Wozniak DF; Department of Psychiatry, Washington University School of Medicine, St. Louis, MO 63110.
  • Vogler CA; Department of Pathology, St. Louis University School of Medicine, St. Louis, MO 63104.
  • Medin JA; Department of Medical Biophysics, University of Toronto, Toronto, ON M5S, Canada.
  • Crawford BE; Pediatrics and Biochemistry, Medical College of Wisconsin, Milwaukee, WI 53226.
  • Sands MS; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.
Proc Natl Acad Sci U S A ; 116(40): 20097-20103, 2019 10 01.
Article en En | MEDLINE | ID: mdl-31527255
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Psicosina / Eliminación de Gen / Ceramidasa Ácida / Leucodistrofia de Células Globoides Límite: Animals / Humans Idioma: En Revista: Proc Natl Acad Sci U S A Año: 2019 Tipo del documento: Article
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Psicosina / Eliminación de Gen / Ceramidasa Ácida / Leucodistrofia de Células Globoides Límite: Animals / Humans Idioma: En Revista: Proc Natl Acad Sci U S A Año: 2019 Tipo del documento: Article