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Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.
Sweeney, Colin L; Pavel-Dinu, Mara; Choi, Uimook; Brault, Julie; Liu, Taylor; Koontz, Sherry; Li, Linhong; Theobald, Narda; Lee, Janet; Bello, Ezekiel A; Wu, Xiaolin; Meis, Ronald J; Dahl, Gary A; Porteus, Matthew H; Malech, Harry L; De Ravin, Suk See.
Afiliación
  • Sweeney CL; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Pavel-Dinu M; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
  • Choi U; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Brault J; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Liu T; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Koontz S; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Li L; MaxCyte Inc., Gaithersburg, MD, USA.
  • Theobald N; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Lee J; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Bello EA; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • Wu X; Cancer Research Technology Program, Leidos Biomedical Research Inc., Frederick, MD, USA.
  • Meis RJ; CELLSCRIPT, LLC, Madison, WI, USA.
  • Dahl GA; CELLSCRIPT, LLC, Madison, WI, USA.
  • Porteus MH; Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA. mporteus@stanford.edu.
  • Malech HL; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
  • De Ravin SS; Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA. sderavin@niaid.nih.gov.
Gene Ther ; 28(6): 373-390, 2021 06.
Article en En | MEDLINE | ID: mdl-33712802
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad Granulomatosa Crónica Límite: Animals / Humans Idioma: En Año: 2021 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad Granulomatosa Crónica Límite: Animals / Humans Idioma: En Año: 2021 Tipo del documento: Article