CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells.

Flynn, Rowan; Grundmann, Alexander; Renz, Peter; Hänseler, Walther; James, William S; Cowley, Sally A; Moore, Michael D

Flynn, Rowan; Grundmann, Alexander; Renz, Peter; Hänseler, Walther; James, William S; Cowley, Sally A; Moore, Michael D.

Afiliação

Flynn R; James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom; Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom.
Grundmann A; Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom.
Renz P; Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom.
Hänseler W; James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom; Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom.
James WS; Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom.
Cowley SA; James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom; Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom.
Moore MD; Sir William Dunn School of Pathology, University of Oxford, Oxford, United Kingdom. Electronic address: kenny.moore@path.ox.ac.uk.

Exp Hematol ; 43(10): 838-848.e3, 2015 Oct.

Article em En | MEDLINE | ID: mdl-26101162

Assuntos

Sistemas CRISPR-Cas; Terapia Genética; Doença Granulomatosa Crônica; Células-Tronco Pluripotentes Induzidas/enzimologia; Glicoproteínas de Membrana/biossíntese; Mutação; NADPH Oxidases/biossíntese; Doença Granulomatosa Crônica/enzimologia; Doença Granulomatosa Crônica/genética; Doença Granulomatosa Crônica/patologia; Doença Granulomatosa Crônica/terapia; Humanos; Células-Tronco Pluripotentes Induzidas/patologia; Íntrons; Glicoproteínas de Membrana/genética; NADPH Oxidase 2; NADPH Oxidases/genética; Explosão Respiratória/genética

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Glicoproteínas de Membrana / Terapia Genética / NADPH Oxidases / Células-Tronco Pluripotentes Induzidas / Sistemas CRISPR-Cas / Doença Granulomatosa Crônica / Mutação Limite: Humans Idioma: En Ano de publicação: 2015 Tipo de documento: Article

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