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. |
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| 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 |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1757-4684 |
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| DOI: | 10.15252/emmm.202115377 |
