Potent and uniform fetal hemoglobin induction via base editing.

Autor: Mayuranathan T; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Newby GA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA., Feng R; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Yao Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Mayberry KD; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Lazzarotto CR; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Li Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Levine RM; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Nimmagadda N; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Dempsey E; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Kang G; Department of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA., Porter SN; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA., Doerfler PA; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Zhang J; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Jang Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Chen J; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Bell HW; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, New South Wales, Australia., Crossley M; School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, New South Wales, Australia., Bhoopalan SV; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Sharma A; Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA., Tisdale JF; Cellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute and National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD, USA., Pruett-Miller SM; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA., Cheng Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Liu DR; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. mitch.weiss@stjude.org., Yen JS; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. jonathan.yen@stjude.org.

Publikováno v: Nature genetics [Nat Genet] 2023 Jul; Vol. 55 (7), pp. 1210-1220. Date of Electronic Publication: 2023 Jul 03.

Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations.

Autor: Woodard KJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.; Integrated Biomedical Sciences Program, College of Graduate Health Sciences, University of Tennessee Health Science Center, Memphis, TN 38163, USA., Doerfler PA; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Mayberry KD; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Sharma A; Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Levine R; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Yen J; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Valentine V; Cytogenetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Palmer LE; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Valentine M; Cytogenetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

Publikováno v: Disease models & mechanisms [Dis Model Mech] 2022 Jun 01; Vol. 15 (6). Date of Electronic Publication: 2022 Jul 06.

Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells.

Autor: Qin K; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Huang P; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Feng R; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Keller CA; Department of Biochemistry and Molecular Biology, Pennsylvania State University, University Park, PA, USA., Peslak SA; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Division of Hematology/Oncology, Department of Medicine, Hospital of the University of Pennsylvania, Philadelphia, PA, USA., Khandros E; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Saari MS; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Lan X; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Systems Biology for Medicine, School of Basic Medical Sciences, Fudan University, Shanghai, China., Mayuranathan T; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Doerfler PA; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Abdulmalik O; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Giardine B; Department of Biochemistry and Molecular Biology, Pennsylvania State University, University Park, PA, USA., Chou ST; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Shi J; Department of Cancer Biology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA., Hardison RC; Department of Biochemistry and Molecular Biology, Pennsylvania State University, University Park, PA, USA., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Blobel GA; Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. blobel@chop.edu.; Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. blobel@chop.edu.

Publikováno v: Nature genetics [Nat Genet] 2022 Jun; Vol. 54 (6), pp. 874-884. Date of Electronic Publication: 2022 May 26.

Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing.

Autor: Leibowitz ML; Howard Hughes Medical Institute, Chevy Chase, MD, USA.; Department of Cell Biology, Blavatnik Institute, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA., Papathanasiou S; Department of Cell Biology, Blavatnik Institute, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA., Doerfler PA; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Blaine LJ; Department of Cell Biology, Blavatnik Institute, Harvard Medical School, Boston, MA, USA.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA., Sun L; Single-Cell Sequencing Program, Dana-Farber Cancer Institute, Boston, MA, USA., Yao Y; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA., Zhang CZ; Department of Biomedical Informatics, Blavatnik Institute, Harvard Medical School, Boston, MA, USA.; Department of Data Sciences, Dana-Farber Cancer Institute, Boston, MA, USA., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. mitch.weiss@stjude.org., Pellman D; Howard Hughes Medical Institute, Chevy Chase, MD, USA. david_pellman@dfci.harvard.edu.; Department of Cell Biology, Blavatnik Institute, Harvard Medical School, Boston, MA, USA. david_pellman@dfci.harvard.edu.; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA, USA. david_pellman@dfci.harvard.edu.

Publikováno v: Nature genetics [Nat Genet] 2021 Jun; Vol. 53 (6), pp. 895-905. Date of Electronic Publication: 2021 Apr 12.

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.