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Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia

Shuqian Xu, Kevin Luk, Qiuming Yao, Anne H. Shen, Jing Zeng, Yuxuan Wu, Hong-Yuan Luo, Christian Brendel, Luca Pinello, David H.K. Chui, Scot A. Wolfe and Daniel E. Bauer

Article Information

PubMed 

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History 
  • Submitted January 3, 2019
  • Accepted January 21, 2019
  • Published online March 4, 2019.

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Contributors 
  • Shuqian Xu, 1 Department of Haematology, Qilu Hospital, Shandong University, Jinan, China;
  • Kevin Luk, 2 Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, United States;
  • Qiuming Yao, 3 Molecular Pathology Unit, Ctr. for Cancer Research, and Ctr. for Computational & Integrative Biology, Massachusetts General Hospital, Department of Pathology, Harvard Medical School, Boston, MA, United States;
  • Anne H. Shen, 4 Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute, Boston, MA, United States;
  • Jing Zeng, 4 Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute, Boston, MA, United States;
  • Yuxuan Wu, 5 Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China;
  • Hong-Yuan Luo, 6 Departments of Medicine, Pathology & Laboratory Medicine, Boston University School of Medicine, Hemoglobin Diagnostic Reference Laboratory, Boston Medical Center, Boston, MA, United States;
  • Christian Brendel, 7 Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA, United States
  • Luca Pinello, 3 Molecular Pathology Unit, Ctr. for Cancer Research, and Ctr. for Computational & Integrative Biology, Massachusetts General Hospital, Department of Pathology, Harvard Medical School, Boston, MA, United States;
  • David H.K. Chui, 6 Departments of Medicine, Pathology & Laboratory Medicine, Boston University School of Medicine, Hemoglobin Diagnostic Reference Laboratory, Boston Medical Center, Boston, MA, United States;
  • Scot A. Wolfe, 2 Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA, United States;
  • Daniel E. Bauer, 4 Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute, Boston, MA, United States;

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