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A small molecule restores function to TRPML1 mutant isoforms responsible for mucolipidosis type IV.

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第一作者： Cheng-Chang,Chen

第一单位： 1] Department of Pharmacy-Center for Drug Research and Center for Integrated Protein Science Munich (CIPSM), Ludwig-Maximilians-Universität München, Munchen 81377, Germany [2].

作者： Cheng-Chang,Chen ^[1] ; Marco,Keller ^[2] ; Martin,Hess ^[3] ; Raphael,Schiffmann ^[4] ; Nicole,Urban ^[5] ; Annette,Wolfgardt ^[6] ; Michael,Schaefer ^[5] ; Franz,Bracher ^[6] ; Martin,Biel ^[7] ; Christian,Wahl-Schott ^[7] ; Christian,Grimm ^[1]

作者单位： 1] Department of Pharmacy-Center for Drug Research and Center for Integrated Protein Science Munich (CIPSM), Ludwig-Maximilians-Universität München, Munchen 81377, Germany [2]. ^[1] 1] Department of Pharmacy, Ludwig-Maximilians-Universität München, Munchen 81377, Germany [2]. ^[2] Department of Biology, Ludwig-Maximilians-Universität München, Munchen 82152, Germany. ^[3] Institute of Metabolic Disease, Baylor Research Institute, Dallas, Texas, USA. ^[4] Rudolf-Boehm-Institute for Pharmacology and Toxicolgy, Universität Leipzig, Leipzig 04107, Germany. ^[5] Department of Pharmacy, Ludwig-Maximilians-Universität München, Munchen 81377, Germany. ^[6] Department of Pharmacy-Center for Drug Research and Center for Integrated Protein Science Munich (CIPSM), Ludwig-Maximilians-Universität München, Munchen 81377, Germany. ^[7]