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BCL11A +58/+55 enhancer-editing facilitates HSPC engraftment and HbF induction in rhesus macaques conditioned with a CD45 antibody-drug conjugate.

Autor: Demirci S; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA. Electronic address: selami.demirci@nih.gov., Zeng J; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA., Palchaudhuri R; Magenta Therapeutics, Cambridge, MA 02139, USA., Wu C; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Abraham DM; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Hayal TB; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Essawi K; Department of Medical Laboratory Technology, College of Applied Medical Sciences, Jazan University, Gizan 45142, Saudi Arabia., Nguyen MA; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA., Stasula U; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Chu R; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Leonard A; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Porter SN; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Khan MBN; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Hinojosa G; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Uchida N; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Hong S; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Lazzarotto CR; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Neri NR; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA., da Silva LF; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA., Pellin D; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA., Verma A; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA., Lanieri L; Magenta Therapeutics, Cambridge, MA 02139, USA., Bhat A; Magenta Therapeutics, Cambridge, MA 02139, USA., Hammond K; Magenta Therapeutics, Cambridge, MA 02139, USA., Tate T; Magenta Therapeutics, Cambridge, MA 02139, USA., Maitland SA; Department of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Sheikhsaran F; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Bonifacino AC; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Krouse AE; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Linde NS; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Engels T; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Golomb J; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Tsai SQ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Pruett-Miller SM; Department of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Scadden DT; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA., Dunbar CE; Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA., Wolfe SA; Department of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Chan Medical School, Worcester, MA 01605, USA., Donahue RE; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA., Olson LM; Magenta Therapeutics, Cambridge, MA 02139, USA., Bauer DE; Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute of MIT and Harvard, Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA. Electronic address: bauer@bloodgroup.tch.harvard.edu., Tisdale JF; National Heart, Lung, and Blood Institute (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20814, USA. Electronic address: johntis@nhlbi.nih.gov.

Publikováno v: Cell stem cell [Cell Stem Cell] 2024 Dec 05. Date of Electronic Publication: 2024 Dec 05.