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Phase I study of liver depot gene therapy in late-onset Pompe disease.
Smith, Edward C; Hopkins, Sam; Case, Laura E; Xu, Ming; Walters, Crista; Dearmey, Stephanie; Han, Sang-Oh; Spears, Tracy G; Chichester, Jessica A; Bossen, Edward H; Hornik, Christoph P; Cohen, Jennifer L; Bali, Deeksha; Kishnani, Priya S; Koeberl, Dwight D.
Afiliação
  • Smith EC; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Hopkins S; Asklepios Biopharmaceutical, Inc. (Askbio), Durham, NC, USA.
  • Case LE; Department of Orthopedics, Duke University School of Medicine, Durham, NC, USA.
  • Xu M; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Walters C; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Dearmey S; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Han SO; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Spears TG; Clinical Trials Statistics, Duke Clinical Research Institute, Durham, NC, USA.
  • Chichester JA; Immunology Core, Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Bossen EH; Department of Pathology, Duke University Medical Center, Durham, NC, USA.
  • Hornik CP; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Cohen JL; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Bali D; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Kishnani PS; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
  • Koeberl DD; Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA. Electronic address: dwight.koeberl@duke.edu.
Mol Ther ; 31(7): 1994-2004, 2023 07 05.
Article em En | MEDLINE | ID: mdl-36805083
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Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II Tipo de estudo: Guideline Limite: Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Bases de dados: MEDLINE Assunto principal: Doença de Depósito de Glicogênio Tipo II Tipo de estudo: Guideline Limite: Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos