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Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.

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Title: Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.
Authors: 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.; Pediatrics and Biochemistry, Medical College of Wisconsin, Milwaukee, WI 53226., Crawford BE; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949., Sands MS; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110; mssands@wustl.edu.; Department of Genetics, Washington University School of Medicine, St. Louis, MO 63110.
Source: Proceedings of the National Academy of Sciences of the United States of America [Proc Natl Acad Sci U S A] 2019 Oct 01; Vol. 116 (40), pp. 20097-20103. Date of Electronic Publication: 2019 Sep 16.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: National Academy of Sciences Country of Publication: United States NLM ID: 7505876 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1091-6490 (Electronic) Linking ISSN: 00278424 NLM ISO Abbreviation: Proc Natl Acad Sci U S A Subsets: MEDLINE
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  Data: Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target.
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  Data: <searchLink fieldCode="AU" term="%22Li+Y%22">Li Y</searchLink>; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.<br /><searchLink fieldCode="AU" term="%22Xu+Y%22">Xu Y</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Benitez+BA%22">Benitez BA</searchLink>; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.<br /><searchLink fieldCode="AU" term="%22Nagree+MS%22">Nagree MS</searchLink>; Department of Medical Biophysics, University of Toronto, Toronto, ON M5S, Canada.<br /><searchLink fieldCode="AU" term="%22Dearborn+JT%22">Dearborn JT</searchLink>; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.<br /><searchLink fieldCode="AU" term="%22Jiang+X%22">Jiang X</searchLink>; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110.<br /><searchLink fieldCode="AU" term="%22Guzman+MA%22">Guzman MA</searchLink>; Department of Pathology, St. Louis University School of Medicine, St. Louis, MO 63104.<br /><searchLink fieldCode="AU" term="%22Woloszynek+JC%22">Woloszynek JC</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Giaramita+A%22">Giaramita A</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Yip+BK%22">Yip BK</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Elsbernd+J%22">Elsbernd J</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Babcock+MC%22">Babcock MC</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Lo+M%22">Lo M</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Fowler+SC%22">Fowler SC</searchLink>; Department of Pharmacology and Toxicology, University of Kansas, Lawrence, KS 66045.<br /><searchLink fieldCode="AU" term="%22Wozniak+DF%22">Wozniak DF</searchLink>; Department of Psychiatry, Washington University School of Medicine, St. Louis, MO 63110.<br /><searchLink fieldCode="AU" term="%22Vogler+CA%22">Vogler CA</searchLink>; Department of Pathology, St. Louis University School of Medicine, St. Louis, MO 63104.<br /><searchLink fieldCode="AU" term="%22Medin+JA%22">Medin JA</searchLink>; Department of Medical Biophysics, University of Toronto, Toronto, ON M5S, Canada.; Pediatrics and Biochemistry, Medical College of Wisconsin, Milwaukee, WI 53226.<br /><searchLink fieldCode="AU" term="%22Crawford+BE%22">Crawford BE</searchLink>; Department of Research, BioMarin Pharmaceutical Inc., Novato, CA 94949.<br /><searchLink fieldCode="AU" term="%22Sands+MS%22">Sands MS</searchLink>; Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110; mssands@wustl.edu.; Department of Genetics, Washington University School of Medicine, St. Louis, MO 63110.
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  Data: <searchLink fieldCode="JN" term="%227505876%22">Proceedings of the National Academy of Sciences of the United States of America</searchLink> [Proc Natl Acad Sci U S A] 2019 Oct 01; Vol. 116 (40), pp. 20097-20103. <i>Date of Electronic Publication: </i>2019 Sep 16.
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