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Lentiviral gene therapy for X-linked chronic granulomatous disease.
Kohn, Donald B; Booth, Claire; Kang, Elizabeth M; Pai, Sung-Yun; Shaw, Kit L; Santilli, Giorgia; Armant, Myriam; Buckland, Karen F; Choi, Uimook; De Ravin, Suk See; Dorsey, Morna J; Kuo, Caroline Y; Leon-Rico, Diego; Rivat, Christine; Izotova, Natalia; Gilmour, Kimberly; Snell, Katie; Dip, Jinhua Xu-Bayford; Darwish, Jinan; Morris, Emma C; Terrazas, Dayna; Wang, Leo D; Bauser, Christopher A; Paprotka, Tobias; Kuhns, Douglas B; Gregg, John; Raymond, Hayley E; Everett, John K; Honnet, Geraldine; Biasco, Luca; Newburger, Peter E; Bushman, Frederic D; Grez, Manuel; Gaspar, H Bobby; Williams, David A; Malech, Harry L; Galy, Anne; Thrasher, Adrian J.
Afiliação
  • Kohn DB; University of California, Los Angeles, CA, USA. dkohn@mednet.ucla.edu.
  • Booth C; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Kang EM; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Pai SY; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Shaw KL; University of California, Los Angeles, CA, USA.
  • Santilli G; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Armant M; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Buckland KF; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Choi U; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • De Ravin SS; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Dorsey MJ; University of California, San Francisco, CA, USA.
  • Kuo CY; University of California, Los Angeles, CA, USA.
  • Leon-Rico D; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Rivat C; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Izotova N; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Gilmour K; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Snell K; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Dip JX; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Darwish J; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Morris EC; University College London Hospitals NHS Foundation Trust, London, UK.
  • Terrazas D; University of California, Los Angeles, CA, USA.
  • Wang LD; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Bauser CA; City of Hope, Beckman Research Institute, Duarte, CA, USA.
  • Paprotka T; Eurofins Genomics Sequencing Europe, Konstanz, Germany.
  • Kuhns DB; Eurofins Genomics Sequencing Europe, Konstanz, Germany.
  • Gregg J; Leidos Biomedical Research, Inc., Frederick National Laboratory for Cancer Research, Frederick, MD, USA.
  • Raymond HE; University of Pennsylvania, Philadelphia, PA, USA.
  • Everett JK; University of Pennsylvania, Philadelphia, PA, USA.
  • Honnet G; University of Pennsylvania, Philadelphia, PA, USA.
  • Biasco L; Genethon, Evry, France.
  • Newburger PE; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Bushman FD; University of Massachusetts Medical School, Worcester, MA, USA.
  • Grez M; University of Pennsylvania, Philadelphia, PA, USA.
  • Gaspar HB; Georg-Speyer Haus, Frankfurt, Germany.
  • Williams DA; Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
  • Malech HL; Orchard Therapeutics, London, UK.
  • Galy A; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Thrasher AJ; Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Nat Med ; 26(2): 200-206, 2020 02.
Article em En | MEDLINE | ID: mdl-31988463

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Lentivirus / Cromossomos Humanos X / Doença Granulomatosa Crônica Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Child / Child, preschool / Humans / Male País/Região como assunto: America do norte / Europa Idioma: En Ano de publicação: 2020 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Lentivirus / Cromossomos Humanos X / Doença Granulomatosa Crônica Tipo de estudo: Clinical_trials Limite: Adolescent / Adult / Child / Child, preschool / Humans / Male País/Região como assunto: America do norte / Europa Idioma: En Ano de publicação: 2020 Tipo de documento: Article