Small Molecule Targets TMED9 and Promotes Lysosomal Degradation to Reverse Proteinopathy.-论文-万方医学网

第一作者： Moran,Dvela-Levitt

第一单位： Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA.

作者： Moran,Dvela-Levitt ^[1] ; Maria,Kost-Alimova ^[2] ; Maheswarareddy,Emani ^[1] ; Eva,Kohnert ^[1] ; Rebecca,Thompson ^[1] ; Eriene-Heidi,Sidhom ^[1] ; Ana,Rivadeneira ^[1] ; Nareh,Sahakian ^[1] ; Julie,Roignot ^[1] ; Gregory,Papagregoriou ^[3] ; Monica S,Montesinos ^[1] ; Abbe R,Clark ^[1] ; David,McKinney ^[2] ; Juan,Gutierrez ^[2] ; Mark,Roth ^[2] ; Lucienne,Ronco ^[2] ; Esther,Elonga ^[2] ; Todd A,Carter ^[2] ; Andreas,Gnirke ^[2] ; Michelle,Melanson ^[2] ; Kate,Hartland ^[2] ; Nicolas,Wieder ^[1] ; Jane C-H,Hsu ^[2] ; Constantinos,Deltas ^[3] ; Rebecca,Hughey ^[4] ; Anthony J,Bleyer ^[5] ; Stanislav,Kmoch ^[5] ; Martina,Živná ^[6] ; Veronika,Barešova ^[6] ; Savithri,Kota ^[7] ; Johannes,Schlondorff ^[7] ; Myriam,Heiman ^[8] ; Seth L,Alper ^[7] ; Florence,Wagner ^[2] ; Astrid,Weins ^[9] ; Todd R,Golub ^[2] ; Eric S,Lander ^[10] ; Anna,Greka ^[11]

作者单位： Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA. ^[1] Broad Institute of MIT and Harvard, Cambridge, MA, USA. ^[2] Molecular Medicine Research Center and Laboratory of Molecular and Medical Genetics, Department of Biological Sciences, University of Cyprus, Nicosia, Cyprus. ^[3] Renal-Electrolyte Division, Department of Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA. ^[4] Section on Nephrology, Wake Forest School of Medicine, Medical Center Blvd., Winston-Salem, NC, USA; Research Unit for Rare Diseases, Department of Pediatrics and Adolescent Medicine, First Faculty of Medicine, Charles University, Prague, Czech Republic. ^[5] Research Unit for Rare Diseases, Department of Pediatrics and Adolescent Medicine, First Faculty of Medicine, Charles University, Prague, Czech Republic. ^[6] Department of Medicine, Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, MA, USA. ^[7] Broad Institute of MIT and Harvard, Cambridge, MA, USA; The Picower Institute for Learning and Memory, Department of Brain and Cognitive Sciences, MIT, Cambridge, MA, USA. ^[8] Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA; Department of Pathology, Brigham and Women's Hospital, Boston, MA, USA. ^[9] Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Biology, MIT, Cambridge, MA, USA; Department of Systems Biology, Harvard Medical School, Boston, MA, USA. ^[10] Broad Institute of MIT and Harvard, Cambridge, MA, USA; Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA. Electronic address: agreka@broadinstitute.org. ^[11]

主题词激活转录因子6(Activating Transcription Factor 6);动物(Animals);苯甲酰胺类(Benzamides);上皮细胞(Epithelial Cells);女(雌)性(Female);移码突变(Frameshift Mutation);庚烷类(Heptanes);人类(Humans);咪唑啉受体(Imidazoline Receptors);诱导多能干细胞(Induced Pluripotent Stem Cells);肾(Kidney);肾疾病(Kidney Diseases);溶酶体(Lysosomes);男(雄)性(Male);小鼠(Mice);小鼠, 转基因(Mice, Transgenic);黏蛋白-1(Mucin-1);RNA干扰(RNA Interference);RNA, 小分子干扰(RNA, Small Interfering);非折叠蛋白质应答(Unfolded Protein Response);膜泡运输蛋白质类(Vesicular Transport Proteins)

DOI 10.1016/j.cell.2019.07.002

PMID 31348885

发布时间 2023-06-07

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Small Molecule Targets TMED9 and Promotes Lysosomal Degradation to Reverse Proteinopathy.

Cell

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Small Molecule Targets TMED9 and Promotes Lysosomal Degradation to Reverse Proteinopathy.

Cell

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