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Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.
Bradbury, Allison M; Bagel, Jessica; Swain, Gary; Miyadera, Keiko; Pesayco, Jill P; Assenmacher, Charles-Antoine; Brisson, Becky; Hendricks, Ian; Wang, Xiao H; Herbst, Zachary; Pyne, Nettie; Odonnell, Patricia; Shelton, G Diane; Gelb, Michael; Hackett, Neil; Szabolcs, Paul; Vite, Charles H; Escolar, Maria.
Afiliação
  • Bradbury AM; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA; Abigail Wexner Research Institute, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43215, USA; Department of Pediatrics, The Ohio State
  • Bagel J; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Swain G; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Miyadera K; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Pesayco JP; Department of Pathology, School of Medicine, University of California San Diego, La Jolla, CA 92161, USA.
  • Assenmacher CA; Comparative Pathology Core, Department of Pathobiology, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Brisson B; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Hendricks I; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Wang XH; Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA 15224, USA.
  • Herbst Z; Department of Chemistry, University of Washington, Seattle, WA 98195, USA.
  • Pyne N; Abigail Wexner Research Institute, Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43215, USA.
  • Odonnell P; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Shelton GD; Department of Pathology, School of Medicine, University of California San Diego, La Jolla, CA 92161, USA.
  • Gelb M; Department of Chemistry, University of Washington, Seattle, WA 98195, USA.
  • Hackett N; Neil Hackett Consulting, New York, NY 10003, USA.
  • Szabolcs P; Department of Pathology, School of Medicine, University of California San Diego, La Jolla, CA 92161, USA.
  • Vite CH; Department of Clinical Sciences and Advanced Medicine, University of Pennsylvania School of Veterinary Medicine, Philadelphia, PA 19104, USA.
  • Escolar M; Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA 15224, USA; Forge Biologics, Grove City, OH 43123, USA.
Mol Ther ; 32(1): 44-58, 2024 Jan 03.
Article em En | MEDLINE | ID: mdl-37952085
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Leucodistrofia de Células Globoides Limite: Animals Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2024 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transplante de Células-Tronco Hematopoéticas / Leucodistrofia de Células Globoides Limite: Animals Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2024 Tipo de documento: Article