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Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy.
Rosen, Steffen; Tiefenbacher, Stefan; Robinson, Mary; Huang, Mei; Srimani, Jaydeep; Mackenzie, Donnie; Christianson, Terri; Pasi, K John; Rangarajan, Savita; Symington, Emily; Giermasz, Adam; Pierce, Glenn F; Kim, Benjamin; Zoog, Stephen J; Vettermann, Christian.
Afiliação
  • Rosen S; Rossix AB, Mölndal, Sweden.
  • Tiefenbacher S; Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO.
  • Robinson M; Colorado Coagulation, Laboratory Corporation of America Holdings, Englewood, CO.
  • Huang M; BioMarin Pharmaceutical Inc, Novato, CA.
  • Srimani J; BioMarin Pharmaceutical Inc, Novato, CA.
  • Mackenzie D; BioMarin Pharmaceutical Inc, Novato, CA.
  • Christianson T; BioMarin Pharmaceutical Inc, Novato, CA.
  • Pasi KJ; Royal London Haemophilia Centre, Barts Health, National Health Service (NHS) Trust, London, United Kingdom.
  • Rangarajan S; University Hospital Southampton, Southampton, United Kingdom.
  • Symington E; Cambridge University Hospitals, NHS Foundation Trust, Cambridge, United Kingdom.
  • Giermasz A; Division of Hematology/Oncology, Department of Medicine, University of California at Davis, Sacramento, CA; and.
  • Pierce GF; Independent Consultant.
  • Kim B; BioMarin Pharmaceutical Inc, Novato, CA.
  • Zoog SJ; BioMarin Pharmaceutical Inc, Novato, CA.
  • Vettermann C; BioMarin Pharmaceutical Inc, Novato, CA.
Blood ; 136(22): 2524-2534, 2020 11 26.
Article em En | MEDLINE | ID: mdl-32915950
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fator VIII / Terapia Genética / Transgenes / Parvovirinae / Hemofilia A Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fator VIII / Terapia Genética / Transgenes / Parvovirinae / Hemofilia A Idioma: En Ano de publicação: 2020 Tipo de documento: Article