Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency.

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第一作者： Lars,Schlotawa

第一单位： Department of Paediatrics and Adolescent Medicine, University Medical Centre Göttingen, Göttingen, Germany.

作者： Lars,Schlotawa ^[1] ; Karolina,Tyka ^[1] ; Matthias,Kettwig ^[1] ; Rebecca C,Ahrens-Nicklas ^[2] ; Matthias,Baud ^[3] ; Tea,Berulava ^[4] ; Nicola,Brunetti-Pierri ^[5] ; Alyssa,Gagne ^[6] ; Zackary M,Herbst ^[7] ; Jean A,Maguire ^[8] ; Jlenia,Monfregola ^[9] ; Tonatiuh,Pena ^[7] ; Karthikeyan,Radhakrishnan ^[8] ; Sophie,Schröder ^[5] ; Elisa A,Waxman ^[6] ; Andrea,Ballabio ^[4] ; Thomas,Dierks ^[10] ; André,Fischer ^[11] ; Deborah L,French ^[4] ; Michael H,Gelb ^[7] ; Jutta,Gärtner ^[8]

作者单位： Department of Paediatrics and Adolescent Medicine, University Medical Centre Göttingen, Göttingen, Germany. ^[1] Division of Human Genetics and Metabolism, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. ^[2] School of Chemistry and Institute for Life Sciences, University of Southampton, Southampton, UK. ^[3] Department for Epigenetics and Systems Medicine in Neurodegenerative Diseases, German Centre for Neurodegenerative Diseases, Göttingen, Germany. ^[4] Telethon Institute of Genetics and Medicine, Pozzuoli, Italy. ^[5] Department of Translational Medicine, University of Naples Federico II, Naples, Italy. ^[6] Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. ^[7] Department of Pathology and Laboratory Medicine, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. ^[8] Department of Chemistry, University of Washington, Seattle, WA, USA. ^[9] Bioinformatics Unit, German Centre for Neurodegenerative Diseases, Göttingen, Germany. ^[10] Faculty of Chemistry, Biochemistry I, Bielefeld University, Bielefeld, Germany. ^[11] Department of Molecular and Human Genetics and Neurological Research Institute, Baylor College of Medicine, Houston, TX, USA. ^[12] Department of Psychiatry and Psychotherapy, University Medical Center Göttingen, Göttingen, Germany. ^[13] Multiscale Bioimaging Cluster of Excellence, University Medical Center Göttingen, University of Göttingen, Göttingen, Germany. ^[14]

关键词 drug screening formylglycine-generating enzyme lysosomal disorder retinoids sulfatase-modifying factor 1

主题词人类(Humans);多种硫酸酯酶缺乏症(Multiple Sulfatase Deficiency Disease);药物评价, 临床前(Drug Evaluation, Preclinical);硫酸酯酶类(Sulfatases);作用于硫族供体的氧化还原酶类(Oxidoreductases Acting on Sulfur Group Donors)

DOI 10.15252/emmm.202114837

PMID 36789546

发布时间 2023-03-21

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准确一般字义偏差语意偏离译文作用小提交