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A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
Nguyen, Giang N; Everett, John K; Kafle, Samita; Roche, Aoife M; Raymond, Hayley E; Leiby, Jacob; Wood, Christian; Assenmacher, Charles-Antoine; Merricks, Elizabeth P; Long, C Tyler; Kazazian, Haig H; Nichols, Timothy C; Bushman, Frederic D; Sabatino, Denise E.
Afiliação
  • Nguyen GN; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Everett JK; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Kafle S; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Roche AM; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Raymond HE; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Leiby J; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Wood C; The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.
  • Assenmacher CA; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Merricks EP; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Long CT; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Kazazian HH; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Nichols TC; UNC Blood Research Center, University of North Carolina School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Bushman FD; Department of Genetic Medicine, Johns Hopkins School of Medicine, Baltimore, MD, USA.
  • Sabatino DE; Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Nat Biotechnol ; 39(1): 47-55, 2021 01.
Article em En | MEDLINE | ID: mdl-33199875
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Fator VIII / Terapia Genética / Dependovirus / Hemofilia A / Fígado Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Animals Idioma: En Revista: Nat Biotechnol Assunto da revista: BIOTECNOLOGIA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Fator VIII / Terapia Genética / Dependovirus / Hemofilia A / Fígado Tipo de estudo: Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Animals Idioma: En Revista: Nat Biotechnol Assunto da revista: BIOTECNOLOGIA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos