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Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome.
Rai, Rajeev; Romito, Marianna; Rivers, Elizabeth; Turchiano, Giandomenico; Blattner, Georges; Vetharoy, Winston; Ladon, Dariusz; Andrieux, Geoffroy; Zhang, Fang; Zinicola, Marta; Leon-Rico, Diego; Santilli, Giorgia; Thrasher, Adrian J; Cavazza, Alessia.
Afiliação
  • Rai R; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Romito M; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Rivers E; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Turchiano G; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Blattner G; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Vetharoy W; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Ladon D; SIHMDS-Acquired Genomics, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
  • Andrieux G; Institute of Medical Bioinformatics and System Medicine, University of Freiburg, 26 Stefan-Meier-Strasse, 79104, Freiburg, Germany.
  • Zhang F; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Zinicola M; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Leon-Rico D; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Santilli G; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Thrasher AJ; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK.
  • Cavazza A; Infection, Immunity and Inflammation Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London, WC1N 1EH, UK. a.cavazza@ucl.ac.uk.
Nat Commun ; 11(1): 4034, 2020 08 12.
Article em En | MEDLINE | ID: mdl-32788576

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Wiskott-Aldrich / Células-Tronco Hematopoéticas / Terapia Genética / Edição de Genes Limite: Animals / Female / Humans / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Síndrome de Wiskott-Aldrich / Células-Tronco Hematopoéticas / Terapia Genética / Edição de Genes Limite: Animals / Female / Humans / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article