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A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency.
Pham, Vi; Sertori Finoti, Livia; Cassidy, Margaret M; Maguire, Jean Ann; Gagne, Alyssa L; Waxman, Elisa A; French, Deborah L; King, Kaitlyn; Zhou, Zitao; Gelb, Michael H; Wongkittichote, Parith; Hong, Xinying; Schlotawa, Lars; Davidson, Beverly L; Ahrens-Nicklas, Rebecca C.
Affiliation
  • Pham V; The Children's Hospital of Philadelphia, Division of Human Genetics and Metabolism, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA; University of Pennsylvania, Perelman School of Medicine, Department of Pediatrics, Philadelphia, PA 19104, USA. Electronic
  • Sertori Finoti L; The Children's Hospital of Philadelphia, Division of Human Genetics and Metabolism, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA. Electronic address: sertorifil@chop.edu.
  • Cassidy MM; The Children's Hospital of Philadelphia, Division of Human Genetics and Metabolism, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA; University of Pennsylvania, Perelman School of Medicine, Department of Pediatrics, Philadelphia, PA 19104, USA. Electronic
  • Maguire JA; The Children's Hospital of Philadelphia, Center for Cellular and Molecular Therapeutics, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA. Electronic address: maguirej@chop.edu.
  • Gagne AL; The Children's Hospital of Philadelphia, Center for Cellular and Molecular Therapeutics, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA. Electronic address: gagnea@chop.edu.
  • Waxman EA; The Children's Hospital of Philadelphia, Center for Cellular and Molecular Therapeutics, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA; Center for Epilepsy and NeuroDevelopmental Disorders (ENDD), The Children's Hospital of Philadelphia, University of Pe
  • French DL; The Children's Hospital of Philadelphia, Center for Cellular and Molecular Therapeutics, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA; Center for Epilepsy and NeuroDevelopmental Disorders (ENDD), The Children's Hospital of Philadelphia, University of Pe
  • King K; The Children's Hospital of Philadelphia, Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address: kingc9@chop.edu.
  • Zhou Z; Department of Chemistry, University of Washington, Seattle, WA 98195, USA.
  • Gelb MH; Department of Chemistry, University of Washington, Seattle, WA 98195, USA. Electronic address: gelb@uw.edu.
  • Wongkittichote P; The Children's Hospital of Philadelphia, Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA; Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
  • Hong X; University of Pennsylvania, Perelman School of Medicine, Department of Pathology and Laboratory Medicine, Philadelphia, PA 19104, USA; The Children's Hospital of Philadelphia, Department of Pathology and Laboratory Medicine, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electroni
  • Schlotawa L; University Medical Center Goettingen, Department of Pediatrics and Adolescent Medicine, Robert-Koch-Str. 40, 37075 Goettingen, Germany; Fraunhofer Institute for Translational Medicine and Pharmacology - Translational Neuroinflammation and Automated Microscopy, Robert-Koch-Str. 40, 37075, Goettingen,
  • Davidson BL; The Children's Hospital of Philadelphia, Center for Cellular and Molecular Therapeutics, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA; University of Pennsylvania, Perelman School of Medicine, Department of Pathology and Laboratory Medicine, Philadelphia
  • Ahrens-Nicklas RC; The Children's Hospital of Philadelphia, Division of Human Genetics and Metabolism, Colket Translational Research Building, 3501 Civic Center Blvd, Philadelphia, PA 19104, USA; University of Pennsylvania, Perelman School of Medicine, Department of Pediatrics, Philadelphia, PA 19104, USA. Electronic
Mol Genet Metab ; 141(2): 108116, 2024 Feb.
Article in En | MEDLINE | ID: mdl-38161139
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Full text: 1 Database: MEDLINE Main subject: Multiple Sulfatase Deficiency Disease / Induced Pluripotent Stem Cells Limits: Humans Language: En Year: 2024 Type: Article
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PubMed Links
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Full text: 1 Database: MEDLINE Main subject: Multiple Sulfatase Deficiency Disease / Induced Pluripotent Stem Cells Limits: Humans Language: En Year: 2024 Type: Article