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) |
References: | Hum Gene Ther. 2013 Feb;24(2):119-31. (PMID: 23330935) Biotechnol Bioeng. 2014 May;111(5):1048-53. (PMID: 23928856) Nature. 2010 Sep 16;467(7313):318-22. (PMID: 20844535) Blood. 2011 Oct 27;118(17):4599-608. (PMID: 21881051) Nature. 2005 Jun 2;435(7042):646-51. (PMID: 15806097) N Engl J Med. 2014 Mar 6;370(10):901-10. (PMID: 24597865) Cell Stem Cell. 2010 Aug 6;7(2):174-85. (PMID: 20619762) Proc Natl Acad Sci U S A. 2003 Dec 9;100(25):14994-9. (PMID: 14630945) Mol Ther. 2006 Jun;13(6):1121-32. (PMID: 16556511) Nat Biotechnol. 2011 Sep;29(9):816-23. (PMID: 21822255) Nat Biotechnol. 2010 Aug;28(8):839-47. (PMID: 20601939) Hematol Oncol Clin North Am. 2014 Apr;28(2):199-216. (PMID: 24589262) Blood. 2011 Nov 10;118(19):5071-9. (PMID: 21885599) Mol Ther. 2013 Sep;21(9):1705-17. (PMID: 23857176) Stem Cells. 2011 Nov;29(11):1717-26. (PMID: 21898685) Medicine (Baltimore). 2005 Nov;84(6):363-76. (PMID: 16267411) Cold Spring Harb Perspect Med. 2013 Oct;3(10):a011783. (PMID: 23813607) Am J Hematol. 2008 Apr;83(4):263-70. (PMID: 17924547) Curr Protoc Immunol. 2008 May;Chapter 15:Unit 15.21. (PMID: 18491294) Nat Biotechnol. 2005 Jan;23(1):69-74. (PMID: 15619619) Biol Blood Marrow Transplant. 2001;7(12):665-73. (PMID: 11787529) Bull World Health Organ. 2008 Jun;86(6):480-7. (PMID: 18568278) Haematologica. 2011 Jan;96(1):13-5. (PMID: 21193430) J Clin Invest. 2013 Jul 1;:null. (PMID: 23863630) Blood. 2014 Mar 6;123(10):1483-6. (PMID: 24429337) Nature. 2014 Jun 12;510(7504):235-40. (PMID: 24870228) Nat Methods. 2011 Sep;8(9):753-5. (PMID: 21765410) Nucleic Acids Res. 2014 Jan;42(2):1365-78. (PMID: 24157834) Oligonucleotides. 2006 Fall;16(3):213-24. (PMID: 16978085) Mol Cell Biol. 1994 Dec;14(12):8096-106. (PMID: 7969147) |
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 |