دورية أكاديمية
Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells.
| العنوان: | Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. |
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| المؤلفون: | Hoban MD; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Cost GJ; Sangamo BioSciences Inc., Richmond, CA;, Mendel MC; Sangamo BioSciences Inc., Richmond, CA;, Romero Z; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Kaufman ML; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Joglekar AV; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Ho M; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Lumaquin D; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Gray D; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Lill GR; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Cooper AR; Molecular Biology Interdepartmental PhD Program, University of California, Los Angeles, Los Angeles, CA;, Urbinati F; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Senadheera S; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Zhu A; Sangamo BioSciences Inc., Richmond, CA;, Liu PQ; Sangamo BioSciences Inc., Richmond, CA;, Paschon DE; Sangamo BioSciences Inc., Richmond, CA;, Zhang L; Sangamo BioSciences Inc., Richmond, CA;, Rebar EJ; Sangamo BioSciences Inc., Richmond, CA;, Wilber A; Department of Medical Microbiology, Immunology and Cell Biology, Southern Illinois University School of Medicine, Springfield, IL;, Wang X; Department of General Internal Medicine and Health Services Research, University of California, Los Angeles, Los Angeles, CA; and., Gregory PD; Sangamo BioSciences Inc., Richmond, CA;, Holmes MC; Sangamo BioSciences Inc., Richmond, CA;, Reik A; Sangamo BioSciences Inc., Richmond, CA;, Hollis RP; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA;, Kohn DB; Department of Microbiology, Immunology, and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, Los Angeles, CA. |
| المصدر: | Blood [Blood] 2015 Apr 23; Vol. 125 (17), pp. 2597-604. Date of Electronic Publication: 2015 Mar 02. |
| نوع المنشور: | Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't |
| اللغة: | English |
| بيانات الدورية: | Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: 2021- : [New York] : Elsevier Original Publication: New York, Grune & Stratton [etc.] |
| مواضيع طبية MeSH: | Genetic Therapy* , Mutation*, Anemia, Sickle Cell/*genetics , Anemia, Sickle Cell/*therapy , Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Anemia, Sickle Cell/pathology ; Animals ; Antigens, CD34/analysis ; Base Sequence ; Bone Marrow Cells/metabolism ; Bone Marrow Cells/pathology ; Cells, Cultured ; Endodeoxyribonucleases/metabolism ; Fetal Blood/transplantation ; Genetic Loci ; Hematopoietic Stem Cell Transplantation ; Hematopoietic Stem Cells/pathology ; Humans ; Mice ; Mice, Inbred NOD ; Mice, SCID ; Molecular Sequence Data ; Zinc Fingers |
| مستخلص: | Sickle cell disease (SCD) is characterized by a single point mutation in the seventh codon of the β-globin gene. Site-specific correction of the sickle mutation in hematopoietic stem cells would allow for permanent production of normal red blood cells. Using zinc-finger nucleases (ZFNs) designed to flank the sickle mutation, we demonstrate efficient targeted cleavage at the β-globin locus with minimal off-target modification. By co-delivering a homologous donor template (either an integrase-defective lentiviral vector or a DNA oligonucleotide), high levels of gene modification were achieved in CD34(+) hematopoietic stem and progenitor cells. Modified cells maintained their ability to engraft NOD/SCID/IL2rγ(null) mice and to produce cells from multiple lineages, although with a reduction in the modification levels relative to the in vitro samples. Importantly, ZFN-driven gene correction in CD34(+) cells from the bone marrow of patients with SCD resulted in the production of wild-type hemoglobin tetramers. (© 2015 by The American Society of Hematology.) |
| التعليقات: | Comment in: Blood. 2015 Apr 23;125(17):2589-90. (PMID: 25907900) |
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| معلومات مُعتمدة: | GM007185 United States GM NIGMS NIH HHS; 5 T32 AI060567 United States AI NIAID NIH HHS; R25 GM055052 United States GM NIGMS NIH HHS; T32 GM007185 United States GM NIGMS NIH HHS; 2P01 HL073104 United States HL NHLBI NIH HHS; T32 AI060567 United States AI NIAID NIH HHS; P01 HL073104 United States HL NHLBI NIH HHS |
| المشرفين على المادة: | 0 (Antigens, CD34) 0 (beta-Globins) EC 3.1.- (Endodeoxyribonucleases) |
| تواريخ الأحداث: | Date Created: 20150304 Date Completed: 20150629 Latest Revision: 20220321 |
| رمز التحديث: | 20240829 |
| مُعرف محوري في PubMed: | PMC4408287 |
| DOI: | 10.1182/blood-2014-12-615948 |
| PMID: | 25733580 |
| قاعدة البيانات: | MEDLINE |
| تدمد: | 1528-0020 |
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| DOI: | 10.1182/blood-2014-12-615948 |