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Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.

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Title: Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.
Authors: Segura EER; Molecular Biology Interdepartmental Doctoral Program, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address: evarosesegura@ucla.edu., Hart K; Department of Human Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Campo Fernandez B; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Brown D; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Tam K; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Gutierrez Garcia A; Department of Biology at California State University, Northridge, Northridge, CA 91330, USA., Seigneurbieux E; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Li K; Department of Molecular, Cell, and Developmental Biology at University of California, Los Angeles, Los Angeles, CA 90095, USA., Mulumba C; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Blakely E; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Masiuk K; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA., Sinha R; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA., Sharma D; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA., Everett J; Department of Microbiology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA 19104, USA., Hogenauer M; Department of Microbiology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA 19104, USA., Cromer MK; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, San Francisco, San Francisco, CA 94143, USA; Center for Maternal-Fetal Precision Medicine at University of California, San Francisco, San Francisco, CA 94143, USA., Bushman F; Department of Microbiology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA 19104, USA., MacKenzie TC; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, San Francisco, San Francisco, CA 94143, USA; Center for Maternal-Fetal Precision Medicine at University of California, San Francisco, San Francisco, CA 94143, USA., Kohn DB; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Pediatrics (Hematology/Oncology), David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address: dkohn1@mednet.ucla.edu.
Source: Cell reports. Medicine [Cell Rep Med] 2025 Oct 21; Vol. 6 (10), pp. 102362. Date of Electronic Publication: 2025 Sep 17.
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101766894 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2666-3791 (Electronic) Linking ISSN: 26663791 NLM ISO Abbreviation: Cell Rep Med Subsets: MEDLINE
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  Data: Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells.
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  Data: <searchLink fieldCode="AU" term="%22Segura+EER%22">Segura EER</searchLink>; Molecular Biology Interdepartmental Doctoral Program, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address: evarosesegura@ucla.edu.<br /><searchLink fieldCode="AU" term="%22Hart+K%22">Hart K</searchLink>; Department of Human Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Campo+Fernandez+B%22">Campo Fernandez B</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Brown+D%22">Brown D</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Tam+K%22">Tam K</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Gutierrez+Garcia+A%22">Gutierrez Garcia A</searchLink>; Department of Biology at California State University, Northridge, Northridge, CA 91330, USA.<br /><searchLink fieldCode="AU" term="%22Seigneurbieux+E%22">Seigneurbieux E</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Li+K%22">Li K</searchLink>; Department of Molecular, Cell, and Developmental Biology at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Mulumba+C%22">Mulumba C</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Blakely+E%22">Blakely E</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Masiuk+K%22">Masiuk K</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.<br /><searchLink fieldCode="AU" term="%22Sinha+R%22">Sinha R</searchLink>; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA.<br /><searchLink fieldCode="AU" term="%22Sharma+D%22">Sharma D</searchLink>; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA.<br /><searchLink fieldCode="AU" term="%22Everett+J%22">Everett J</searchLink>; Department of Microbiology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Hogenauer+M%22">Hogenauer M</searchLink>; Department of Microbiology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22Cromer+MK%22">Cromer MK</searchLink>; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, San Francisco, San Francisco, CA 94143, USA; Center for Maternal-Fetal Precision Medicine at University of California, San Francisco, San Francisco, CA 94143, USA.<br /><searchLink fieldCode="AU" term="%22Bushman+F%22">Bushman F</searchLink>; Department of Microbiology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA 19104, USA.<br /><searchLink fieldCode="AU" term="%22MacKenzie+TC%22">MacKenzie TC</searchLink>; Department of Surgery, Division of Pediatric Surgery at University of California, San Francisco, San Francisco, CA 94143, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, San Francisco, San Francisco, CA 94143, USA; Center for Maternal-Fetal Precision Medicine at University of California, San Francisco, San Francisco, CA 94143, USA.<br /><searchLink fieldCode="AU" term="%22Kohn+DB%22">Kohn DB</searchLink>; Department of Microbiology, Immunology & Molecular Genetics, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Department of Pediatrics (Hematology/Oncology), David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA; Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address: dkohn1@mednet.ucla.edu.
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