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

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Bibliographic Details
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
Database: MEDLINE Ultimate
Description
ISSN:2666-3791
DOI:10.1016/j.xcrm.2025.102362