Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.

Autor: Métais JY; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Doerfler PA; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Mayuranathan T; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Bauer DE; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA.; Department of Pediatrics, Harvard Medical School, Boston, MA.; Harvard Stem Cell Institute, Cambridge, MA.; Broad Institute of MIT and Harvard, Cambridge, MA., Fowler SC; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Hsieh MM; Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD., Katta V; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Keriwala S; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Lazzarotto CR; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Luk K; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA; and., Neel MD; Division of Orthopedics., Perry SS; Flow Cytometry and Cell Sorting Shared Resource., Peters ST; Department of Cell and Molecular Biology, and., Porter SN; Department of Cell and Molecular Biology, and., Ryu BY; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Sharma A; Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN., Shea D; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA., Tisdale JF; Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD., Uchida N; Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD., Wolfe SA; Department of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA; and., Woodard KJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Wu Y; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA., Yao Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Zeng J; Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA., Pruett-Miller S; Department of Cell and Molecular Biology, and., Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN.

Publikováno v: Blood advances [Blood Adv] 2019 Nov 12; Vol. 3 (21), pp. 3379-3392.