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Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies.
Breda, Laura; Ghiaccio, Valentina; Tanaka, Naoto; Jarocha, Danuta; Ikawa, Yasuhiro; Abdulmalik, Osheiza; Dong, Alisa; Casu, Carla; Raabe, Tobias D; Shan, Xiaochuan; Danet-Desnoyers, Gwenn A; Doto, Aoife M; Everett, John; Bushman, Frederic D; Radaelli, Enrico; Assenmacher, Charles A; Tarrant, James C; Hoepp, Natalie; Kurita, Ryo; Nakamura, Yukio; Guzikowski, Virginia; Smith-Whitley, Kim; Kwiatkowski, Janet L; Rivella, Stefano.
Afiliação
  • Breda L; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: bredal@email.chop.edu.
  • Ghiaccio V; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Tanaka N; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Jarocha D; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Ikawa Y; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Abdulmalik O; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Dong A; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Casu C; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Raabe TD; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Shan X; Stem and Xenograft Core, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Danet-Desnoyers GA; Stem and Xenograft Core, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Doto AM; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Everett J; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Bushman FD; Department of Microbiology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Radaelli E; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Assenmacher CA; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Tarrant JC; Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Hoepp N; Clinical Pathology Laboratory, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Kurita R; RIKEN BioResource Center, Tsukuba, Ibaraki, Japan.
  • Nakamura Y; RIKEN BioResource Center, Tsukuba, Ibaraki, Japan.
  • Guzikowski V; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
  • Smith-Whitley K; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Kwiatkowski JL; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Rivella S; Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA; Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; Cell and Molecular Biology Affinity Group (CAMB), University of Pennsylvania, Philadelphia, PA, USA; Ra
Mol Ther ; 29(4): 1625-1638, 2021 04 07.
Article em En | MEDLINE | ID: mdl-33515514
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Transplante de Medula Óssea / Talassemia beta / Globinas beta / Anemia Falciforme Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Transplante de Medula Óssea / Talassemia beta / Globinas beta / Anemia Falciforme Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2021 Tipo de documento: Article