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Targeted Repair of p47-CGD in iPSCs by CRISPR/Cas9: Functional Correction without Cleavage in the Highly Homologous Pseudogenes.
Klatt, Denise; Cheng, Erica; Philipp, Friederike; Selich, Anton; Dahlke, Julia; Schmidt, Reinhold E; Schott, Juliane W; Büning, Hildegard; Hoffmann, Dirk; Thrasher, Adrian J; Schambach, Axel.
Afiliação
  • Klatt D; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
  • Cheng E; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
  • Philipp F; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany; Fraunhofer Institute for Toxicology and Experimental Medicine, 30625 Hannover, Germany.
  • Selich A; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany.
  • Dahlke J; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
  • Schmidt RE; Department of Immunology and Rheumatology, Hannover Medical School, 30625 Hannover, Germany.
  • Schott JW; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany.
  • Büning H; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
  • Hoffmann D; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany.
  • Thrasher AJ; Infection, Immunity and Inflammation Program, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK; Great Ormond Street Hospital NHS Foundation Trust, London WC1N 1EH, UK.
  • Schambach A; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Strasse 1, 30625 Hannover, Germany; REBIRTH Cluster of Excellence, Hannover Medical School, 30625 Hannover, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
Stem Cell Reports ; 13(4): 590-598, 2019 10 08.
Article em En | MEDLINE | ID: mdl-31543470
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: NADPH Oxidases / Células-Tronco Pluripotentes Induzidas / Sistemas CRISPR-Cas / Edição de Genes / Doença Granulomatosa Crônica Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article
Texto completo
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PubMed Links
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: NADPH Oxidases / Células-Tronco Pluripotentes Induzidas / Sistemas CRISPR-Cas / Edição de Genes / Doença Granulomatosa Crônica Tipo de estudo: Prognostic_studies Limite: Humans Idioma: En Ano de publicação: 2019 Tipo de documento: Article