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Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Schejtman, Andrea; Vetharoy, Winston; Choi, Uimook; Rivat, Christine; Theobald, Narda; Piras, Giuseppa; Leon-Rico, Diego; Buckland, Karen; Armenteros-Monterroso, Elena; Benedetti, Sara; Ashworth, Michael T; Rothe, Michael; Schambach, Axel; Gaspar, Hubert Bobby; Kang, Elizabeth M; Malech, Harry L; Thrasher, Adrian J; Santilli, Giorgia.
Afiliación
  • Schejtman A; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
  • Vetharoy W; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
  • Choi U; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
  • Rivat C; Great Ormond Street Hospital for Children, NHS Foundation Trust, London, United Kingdom.
  • Theobald N; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
  • Piras G; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
  • Leon-Rico D; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
  • Buckland K; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
  • Armenteros-Monterroso E; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
  • Benedetti S; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
  • Ashworth MT; Department of Histopathology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, United Kingdom.
  • Rothe M; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
  • Schambach A; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.
  • Gaspar HB; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Kang EM; Orchard Therapeutics, London, United Kingdom.
  • Malech HL; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
  • Thrasher AJ; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
  • Santilli G; Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Hum Gene Ther ; 32(17-18): 949-958, 2021 09.
Article en En | MEDLINE | ID: mdl-33740872
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad Granulomatosa Crónica Tipo de estudio: Guideline Límite: Animals / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2021 Tipo del documento: Article País de afiliación: Reino Unido
Texto completo
Añadir a Mi BVS
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad Granulomatosa Crónica Tipo de estudio: Guideline Límite: Animals / Humans Idioma: En Revista: Hum Gene Ther Asunto de la revista: GENETICA MEDICA / TERAPEUTICA Año: 2021 Tipo del documento: Article País de afiliación: Reino Unido