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TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann-Pick type C1, and Batten disease.

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Title: TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann-Pick type C1, and Batten disease.
Authors: Scotto Rosato A; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany., Krogsaeter EK; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany., Jaślan D; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany., Abrahamian C; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany., Montefusco S; Telethon Institute of Genetics and Medicine, Naples, Italy., Soldati C; Telethon Institute of Genetics and Medicine, Naples, Italy., Spix B; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany., Pizzo MT; Telethon Institute of Genetics and Medicine, Naples, Italy., Grieco G; Telethon Institute of Genetics and Medicine, Naples, Italy., Böck J; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany., Wyatt A; Experimental Pharmacology, Center for Molecular Signaling (PZMS), Saarland University School of Medicine, Homburg, Germany., Wünkhaus D; Evotec AG, Hamburg, Germany., Passon M; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany., Stieglitz M; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany., Keller M; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany., Hermey G; Center for Molecular Neurobiology Hamburg (ZMNH), Institute of Molecular and Cellular Cognition, UKE, Hamburg, Germany., Markmann S; Evotec AG, Hamburg, Germany., Gruber-Schoffnegger D; Evotec AG, Hamburg, Germany., Cotman S; Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA., Johannes L; Cellular and Chemical Biology Department, Institut Curie, U1143 INSERM, UMR3666 CNRS, PSL Research University, Paris, France., Crusius D; Institute for Stroke and Dementia Research (ISD), Ludwig-Maximilians-University (LMU) Hospital, Munich, Germany., Boehm U; Experimental Pharmacology, Center for Molecular Signaling (PZMS), Saarland University School of Medicine, Homburg, Germany., Wahl-Schott C; Institute for Neurophysiology, Hannover Medical School, Hannover, Germany., Biel M; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany., Bracher F; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany., De Leonibus E; Telethon Institute of Genetics and Medicine, Naples, Italy.; Institute of Biochemistry and Cell Biology (IBBC), CNR, Rome, Italy., Polishchuk E; Telethon Institute of Genetics and Medicine, Naples, Italy., Medina DL; Telethon Institute of Genetics and Medicine, Naples, Italy.; Medical Genetics Unit, Department of Medical and Translational Science, Federico II University, Naples, Italy., Paquet D; Institute for Stroke and Dementia Research (ISD), Ludwig-Maximilians-University (LMU) Hospital, Munich, Germany.; Munich Cluster for Systems Neurology (SyNergy), Ludwig-Maximilians-University (LMU), Munich, Germany., Grimm C; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.
Source: EMBO molecular medicine [EMBO Mol Med] 2022 Sep 07; Vol. 14 (9), pp. e15377. Date of Electronic Publication: 2022 Aug 05.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: EMBO Press Country of Publication: Germany NLM ID: 101487380 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1757-4684 (Electronic) Linking ISSN: 17574676 NLM ISO Abbreviation: EMBO Mol Med Subsets: MEDLINE
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  Data: TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann-Pick type C1, and Batten disease.
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  Data: <searchLink fieldCode="AU" term="%22Scotto+Rosato+A%22">Scotto Rosato A</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Krogsaeter+EK%22">Krogsaeter EK</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Jaślan+D%22">Jaślan D</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Abrahamian+C%22">Abrahamian C</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Montefusco+S%22">Montefusco S</searchLink>; Telethon Institute of Genetics and Medicine, Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Soldati+C%22">Soldati C</searchLink>; Telethon Institute of Genetics and Medicine, Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Spix+B%22">Spix B</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Pizzo+MT%22">Pizzo MT</searchLink>; Telethon Institute of Genetics and Medicine, Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Grieco+G%22">Grieco G</searchLink>; Telethon Institute of Genetics and Medicine, Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Böck+J%22">Böck J</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Wyatt+A%22">Wyatt A</searchLink>; Experimental Pharmacology, Center for Molecular Signaling (PZMS), Saarland University School of Medicine, Homburg, Germany.<br /><searchLink fieldCode="AU" term="%22Wünkhaus+D%22">Wünkhaus D</searchLink>; Evotec AG, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Passon+M%22">Passon M</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Stieglitz+M%22">Stieglitz M</searchLink>; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Keller+M%22">Keller M</searchLink>; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Hermey+G%22">Hermey G</searchLink>; Center for Molecular Neurobiology Hamburg (ZMNH), Institute of Molecular and Cellular Cognition, UKE, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Markmann+S%22">Markmann S</searchLink>; Evotec AG, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Gruber-Schoffnegger+D%22">Gruber-Schoffnegger D</searchLink>; Evotec AG, Hamburg, Germany.<br /><searchLink fieldCode="AU" term="%22Cotman+S%22">Cotman S</searchLink>; Department of Neurology, Center for Genomic Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Johannes+L%22">Johannes L</searchLink>; Cellular and Chemical Biology Department, Institut Curie, U1143 INSERM, UMR3666 CNRS, PSL Research University, Paris, France.<br /><searchLink fieldCode="AU" term="%22Crusius+D%22">Crusius D</searchLink>; Institute for Stroke and Dementia Research (ISD), Ludwig-Maximilians-University (LMU) Hospital, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Boehm+U%22">Boehm U</searchLink>; Experimental Pharmacology, Center for Molecular Signaling (PZMS), Saarland University School of Medicine, Homburg, Germany.<br /><searchLink fieldCode="AU" term="%22Wahl-Schott+C%22">Wahl-Schott C</searchLink>; Institute for Neurophysiology, Hannover Medical School, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Biel+M%22">Biel M</searchLink>; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Bracher+F%22">Bracher F</searchLink>; Department of Pharmacy, Center for Drug Research, Ludwig-Maximilians-Universität, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22De+Leonibus+E%22">De Leonibus E</searchLink>; Telethon Institute of Genetics and Medicine, Naples, Italy.; Institute of Biochemistry and Cell Biology (IBBC), CNR, Rome, Italy.<br /><searchLink fieldCode="AU" term="%22Polishchuk+E%22">Polishchuk E</searchLink>; Telethon Institute of Genetics and Medicine, Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Medina+DL%22">Medina DL</searchLink>; Telethon Institute of Genetics and Medicine, Naples, Italy.; Medical Genetics Unit, Department of Medical and Translational Science, Federico II University, Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Paquet+D%22">Paquet D</searchLink>; Institute for Stroke and Dementia Research (ISD), Ludwig-Maximilians-University (LMU) Hospital, Munich, Germany.; Munich Cluster for Systems Neurology (SyNergy), Ludwig-Maximilians-University (LMU), Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Grimm+C%22">Grimm C</searchLink>; Faculty of Medicine, Walther Straub Institute of Pharmacology and Toxicology, Ludwig-Maximilians-Universität, Munich, Germany.
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