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Development of ß-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi, Annalisa; Camarena, Joab; Lahiri, Premanjali; Segal, Helen; Srifa, Waracharee; Vakulskas, Christopher A; Frock, Richard L; Kenrick, Josefin; Lee, Ciaran; Talbott, Narae; Skowronski, Jason; Cromer, M Kyle; Charlesworth, Carsten T; Bak, Rasmus O; Mantri, Sruthi; Bao, Gang; DiGiusto, David; Tisdale, John; Wright, J Fraser; Bhatia, Neehar; Roncarolo, Maria Grazia; Dever, Daniel P; Porteus, Matthew H.
Afiliação
  • Lattanzi A; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Camarena J; Center for Definitive and Curative Medicine, Stanford University, Stanford, CA 94305, USA.
  • Lahiri P; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Segal H; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
  • Srifa W; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
  • Vakulskas CA; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Frock RL; Integrated DNA Technologies Inc., Coralville, IA 52241, USA.
  • Kenrick J; Department of Radiation Oncology, Stanford University, Stanford, CA 94305, USA.
  • Lee C; Department of Radiation Oncology, Stanford University, Stanford, CA 94305, USA.
  • Talbott N; APC Microbiome Ireland, University College Cork, T12 YN60 Cork, Ireland.
  • Skowronski J; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
  • Cromer MK; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
  • Charlesworth CT; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Bak RO; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Mantri S; Department of Biomedicine, Aarhus University, DK-8000 Aarhus, Denmark.
  • Bao G; Aarhus Institute of Advanced Studies (AIAS), Aarhus University, DK-8000 Aarhus, Denmark.
  • DiGiusto D; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Tisdale J; Department of Bioengineering, Rice University, Houston, TX 77006, USA.
  • Wright JF; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
  • Bhatia N; Molecular and Clinical Hematology Branch, NHLBI, Bethesda, MD 20814, USA.
  • Roncarolo MG; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Dever DP; Center for Definitive and Curative Medicine, Stanford University, Stanford, CA 94305, USA.
  • Porteus MH; Laboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
Sci Transl Med ; 13(598)2021 06 16.
Article em En | MEDLINE | ID: mdl-34135108

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Compostos Heterocíclicos / Anemia Falciforme Limite: Animals / Humans Idioma: En Revista: Sci Transl Med Assunto da revista: CIENCIA / MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Compostos Heterocíclicos / Anemia Falciforme Limite: Animals / Humans Idioma: En Revista: Sci Transl Med Assunto da revista: CIENCIA / MEDICINA Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Estados Unidos