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Highly efficient editing of the ß-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Park, So Hyun; Lee, Ciaran M; Dever, Daniel P; Davis, Timothy H; Camarena, Joab; Srifa, Waracharee; Zhang, Yankai; Paikari, Alireza; Chang, Alicia K; Porteus, Matthew H; Sheehan, Vivien A; Bao, Gang.
Affiliation
  • Park SH; Department of Bioengineering, Rice University, Houston, TX 77030, USA.
  • Lee CM; Department of Bioengineering, Rice University, Houston, TX 77030, USA.
  • Dever DP; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Davis TH; Department of Bioengineering, Rice University, Houston, TX 77030, USA.
  • Camarena J; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Srifa W; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Zhang Y; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Paikari A; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Chang AK; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Porteus MH; Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  • Sheehan VA; Texas Children's Hematology Center, Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.
  • Bao G; Department of Bioengineering, Rice University, Houston, TX 77030, USA.
Nucleic Acids Res ; 47(15): 7955-7972, 2019 09 05.
Article in En | MEDLINE | ID: mdl-31147717
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cells / Hematopoietic Stem Cell Transplantation / Beta-Globins / Gene Editing / Anemia, Sickle Cell Limits: Animals / Humans Language: En Journal: Nucleic Acids Res Year: 2019 Document type: Article Affiliation country: United States Country of publication: United kingdom
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cells / Hematopoietic Stem Cell Transplantation / Beta-Globins / Gene Editing / Anemia, Sickle Cell Limits: Animals / Humans Language: En Journal: Nucleic Acids Res Year: 2019 Document type: Article Affiliation country: United States Country of publication: United kingdom