CRISPR/Cas9 ß-globin gene targeting in human haematopoietic stem cells.

Dever, Daniel P; Bak, Rasmus O; Reinisch, Andreas; Camarena, Joab; Washington, Gabriel; Nicolas, Carmencita E; Pavel-Dinu, Mara; Saxena, Nivi; Wilkens, Alec B; Mantri, Sruthi; Uchida, Nobuko; Hendel, Ayal; Narla, Anupama; Majeti, Ravindra; Weinberg, Kenneth I; Porteus, Matthew H

Dever, Daniel P; Bak, Rasmus O; Reinisch, Andreas; Camarena, Joab; Washington, Gabriel; Nicolas, Carmencita E; Pavel-Dinu, Mara; Saxena, Nivi; Wilkens, Alec B; Mantri, Sruthi; Uchida, Nobuko; Hendel, Ayal; Narla, Anupama; Majeti, Ravindra; Weinberg, Kenneth I; Porteus, Matthew H.

Afiliação

Dever DP; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Bak RO; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Reinisch A; Department of Medicine, Division of Hematology, Cancer Institute, and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, California 94305, USA.
Camarena J; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Washington G; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Nicolas CE; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Pavel-Dinu M; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Saxena N; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Wilkens AB; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Mantri S; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Uchida N; Stem Cells, Inc. 7707 Gateway Blvd., Suite 140, Newark, California 94560, USA.
Hendel A; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Narla A; Division of Hematology/Oncology, Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94035, USA.
Majeti R; Department of Medicine, Division of Hematology, Cancer Institute, and Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, California 94305, USA.
Weinberg KI; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.
Porteus MH; Department of Pediatrics, Stanford University, Stanford, California 94305, USA.

Nature ; 539(7629): 384-389, 2016 11 17.

Article em En | MEDLINE | ID: mdl-27820943

Assuntos

Anemia Falciforme/genética; Sistemas CRISPR-Cas/genética; Edição de Genes/métodos; Marcação de Genes; Terapia Genética/métodos; Células-Tronco Hematopoéticas/metabolismo; Globinas beta/genética; Alelos; Anemia Falciforme/patologia; Anemia Falciforme/terapia; Animais; Antígenos CD34/metabolismo; Proteínas Associadas a CRISPR/metabolismo; Diferenciação Celular; Linhagem da Célula; Separação Celular; Dependovirus/genética; Eritrócitos; Feminino; Citometria de Fluxo; Genes Reporter; Recombinação Homóloga; Humanos; Imãs; Camundongos Endogâmicos NOD; Camundongos SCID; Microesferas; Mutação; RNA Mensageiro/biossíntese; RNA Mensageiro/genética; Talassemia beta/genética; Talassemia beta/terapia

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Células-Tronco Hematopoéticas / Terapia Genética / Marcação de Genes / Globinas beta / Sistemas CRISPR-Cas / Edição de Genes / Anemia Falciforme Limite: Animals / Female / Humans Idioma: En Ano de publicação: 2016 Tipo de documento: Article

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