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Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep.
Nelvagal, Hemanth R; Eaton, Samantha L; Wang, Sophie H; Eultgen, Elizabeth M; Takahashi, Keigo; Le, Steven Q; Nesbitt, Rachel; Dearborn, Joshua T; Siano, Nicholas; Puhl, Ana C; Dickson, Patricia I; Thompson, Gerard; Murdoch, Fraser; Brennan, Paul M; Gray, Mark; Greenhalgh, Stephen N; Tennant, Peter; Gregson, Rachael; Clutton, Eddie; Nixon, James; Proudfoot, Chris; Guido, Stefano; Lillico, Simon G; Whitelaw, C Bruce A; Lu, Jui-Yun; Hofmann, Sandra L; Ekins, Sean; Sands, Mark S; Wishart, Thomas M; Cooper, Jonathan D.
Afiliação
  • Nelvagal HR; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.
  • Eaton SL; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Wang SH; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.
  • Eultgen EM; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.
  • Takahashi K; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.
  • Le SQ; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.
  • Nesbitt R; Department of Medicine, Washington University in St. Louis, School of Medicine, St .Louis, Missouri, USA.
  • Dearborn JT; Department of Medicine, Washington University in St. Louis, School of Medicine, St .Louis, Missouri, USA.
  • Siano N; Discovery Science Division, Amicus Therapeutics Inc., Philadelphia, Pennsylvania, USA.
  • Puhl AC; Collaborations Pharmaceuticals Inc., Lab 3510, Raleigh, North Carolina, USA.
  • Dickson PI; Department of Pediatrics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.
  • Thompson G; Department of Genetics, Washington University in St. Louis, School of Medicine, St. Louis, Missouri, USA.
  • Murdoch F; Centre for Clinical Brain Sciences, University of Edinburgh, Chancellor's Building, Edinburgh, Scotland, United Kingdom.
  • Brennan PM; Department of Clinical Neurosciences, NHS Lothian, Edinburgh, Scotland, United Kingdom.
  • Gray M; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Greenhalgh SN; Centre for Clinical Brain Sciences, University of Edinburgh, Chancellor's Building, Edinburgh, Scotland, United Kingdom.
  • Tennant P; Department of Clinical Neurosciences, NHS Lothian, Edinburgh, Scotland, United Kingdom.
  • Gregson R; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Clutton E; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Nixon J; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Proudfoot C; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Guido S; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Lillico SG; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Whitelaw CBA; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Lu JY; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Hofmann SL; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Ekins S; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Sands MS; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Wishart TM; The Large Animal Research and Imaging Facility (LARIF), Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
  • Cooper JD; The Roslin Institute and Royal (Dick) School of Veterinary Studies, University of Edinburgh, Easter Bush Campus, Easter Bush, Scotland, United Kingdom.
J Clin Invest ; 132(20)2022 10 17.
Article em En | MEDLINE | ID: mdl-36040802
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Lipofuscinoses Ceroides Neuronais Tipo de estudo: Prognostic_studies Limite: Animals / Child / Humans Idioma: En Revista: J Clin Invest Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Lipofuscinoses Ceroides Neuronais Tipo de estudo: Prognostic_studies Limite: Animals / Child / Humans Idioma: En Revista: J Clin Invest Ano de publicação: 2022 Tipo de documento: Article País de afiliação: Estados Unidos