دورية أكاديمية
أعرض في EDS

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.

التفاصيل البيبلوغرافية
العنوان: CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells.
المؤلفون: Hoban MD; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Lumaquin D; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Kuo CY; Division of Allergy and Immunology, Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, California, USA., Romero Z; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Long J; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA.; Biology Department, California State University, Northridge, California, USA., Ho M; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Young CS; Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, California, USA.; Molecular Biology Interdepartmental PhD Program (MBIDP), University of California, Los Angeles, California, USA., Mojadidi M; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Fitz-Gibbon S; Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles, California, USA.; Institute for Genomics and Proteomics, University of California, Los Angeles, California, USA., Cooper AR; Molecular Biology Interdepartmental PhD Program (MBIDP), University of California, Los Angeles, California, USA., Lill GR; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Urbinati F; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Campo-Fernandez B; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Bjurstrom CF; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Pellegrini M; Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles, California, USA.; Institute for Genomics and Proteomics, University of California, Los Angeles, California, USA., Hollis RP; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California USA.; Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research, University of California, Los Angeles, California, USA.
المصدر: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2016 Sep; Vol. 24 (9), pp. 1561-9. Date of Electronic Publication: 2016 Jul 29.
نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
اللغة: English
بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
أسماء مطبوعة: Publication: 2017- : Cambridge, MA : Cell Press
Original Publication: San Diego, CA : Academic Press, 2000-
مواضيع طبية MeSH: CRISPR-Cas Systems* , Gene Editing* , Mutation* , Targeted Gene Repair*, Anemia, Sickle Cell/*genetics , Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Anemia, Sickle Cell/therapy ; Base Sequence ; Cell Line ; DNA Cleavage ; Gene Targeting ; Genetic Loci ; Humans ; Protein Binding ; RNA, Guide, CRISPR-Cas Systems ; Transcription Activator-Like Effector Nucleases/metabolism
مستخلص: Targeted genome editing technology can correct the sickle cell disease mutation of the β-globin gene in hematopoietic stem cells. This correction supports production of red blood cells that synthesize normal hemoglobin proteins. Here, we demonstrate that Transcription Activator-Like Effector Nucleases (TALENs) and the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system can target DNA sequences around the sickle-cell mutation in the β-globin gene for site-specific cleavage and facilitate precise correction when a homologous donor template is codelivered. Several pairs of TALENs and multiple CRISPR guide RNAs were evaluated for both on-target and off-target cleavage rates. Delivery of the CRISPR/Cas9 components to CD34+ cells led to over 18% gene modification in vitro. Additionally, we demonstrate the correction of the sickle cell disease mutation in bone marrow derived CD34+ hematopoietic stem and progenitor cells from sickle cell disease patients, leading to the production of wild-type hemoglobin. These results demonstrate correction of the sickle mutation in patient-derived CD34+ cells using CRISPR/Cas9 technology.
References: Nat Biotechnol. 2015 Dec;33(12):1256-1263. (PMID: 26551060)
Nat Biotechnol. 2015 Sep;33(9):985-9. (PMID: 26121415)
JAMA. 2014 Jul 2;312(1):48-56. (PMID: 25058217)
Nat Methods. 2014 Apr;11(4):399-402. (PMID: 24584192)
N Engl J Med. 2014 Mar 6;370(10):901-10. (PMID: 24597865)
Cell. 2013 Sep 12;154(6):1380-9. (PMID: 23992846)
Cell Stem Cell. 2010 Aug 6;7(2):174-85. (PMID: 20619762)
Nat Biotechnol. 2014 Mar;32(3):279-84. (PMID: 24463574)
Trends Biotechnol. 2013 Jul;31(7):397-405. (PMID: 23664777)
Mol Ther. 2006 Jun;13(6):1121-32. (PMID: 16556511)
Genome Res. 2012 Mar;22(3):539-48. (PMID: 22183967)
Curr Protoc Stem Cell Biol. 2016 Feb 03;36:5B.4.1-10. (PMID: 26840227)
Curr Opin Chem Biol. 2012 Aug;16(3-4):268-77. (PMID: 22819644)
Nat Biotechnol. 2015 Feb;33(2):175-8. (PMID: 25599175)
Nucleic Acids Res. 2013 Nov;41(20):9584-92. (PMID: 23939622)
Genome Res. 2013 Jul;23(7):1182-93. (PMID: 23568838)
Nat Biotechnol. 2011 Aug 07;29(9):816-23. (PMID: 21822255)
Methods Enzymol. 2014;546:47-78. (PMID: 25398335)
Genome Res. 2014 Jan;24(1):132-41. (PMID: 24253446)
Haematologica. 2011 Jan;96(1):128-33. (PMID: 20935000)
Blood. 2015 Apr 23;125(17):2597-604. (PMID: 25733580)
Nat Biotechnol. 2015 May;33(5):538-42. (PMID: 25798939)
Proc Natl Acad Sci U S A. 2009 Jun 30;106(26):10620-5. (PMID: 19549848)
Nature. 2015 Oct 15;526(7573):351-60. (PMID: 26469046)
Nat Biotechnol. 2014 Jun;32(6):569-76. (PMID: 24770325)
Nat Biotechnol. 2015 Feb;33(2):187-97. (PMID: 25513782)
Biol Blood Marrow Transplant. 2008 Nov;14 (11):1270-8. (PMID: 18940682)
Nat Biotechnol. 2005 Jan;23(1):69-74. (PMID: 15619619)
Br J Haematol. 2007 Nov;139(3):504-7. (PMID: 17910640)
Nat Biotechnol. 2013 Sep;31(9):807-9. (PMID: 24022156)
J Clin Invest. 2013 Jul 1;:null. (PMID: 23863630)
Nature. 2014 Jun 12;510(7504):235-40. (PMID: 24870228)
Nat Biotechnol. 2013 Sep;31(9):822-6. (PMID: 23792628)
Nat Protoc. 2013 Nov;8(11):2281-308. (PMID: 24157548)
Nucleic Acids Res. 2014 Jan;42(2):1365-78. (PMID: 24157834)
Nat Biotechnol. 2007 Jul;25(7):786-93. (PMID: 17603476)
Nat Methods. 2014 Jul;11(7):712. (PMID: 25110782)
Nucleic Acids Res. 2014 Jun;42(11):7473-85. (PMID: 24838573)
معلومات مُعتمدة: R25 GM055052 United States GM NIGMS NIH HHS; P30 AI028697 United States AI NIAID NIH HHS; T32 GM007185 United States GM NIGMS NIH HHS; 2013158 United States DDCF_ Doris Duke Charitable Foundation; T32 AI060567 United States AI NIAID NIH HHS; P01 HL073104 United States HL NHLBI NIH HHS
المشرفين على المادة: 0 (RNA, Guide, CRISPR-Cas Systems)
0 (beta-Globins)
EC 3.1.- (Transcription Activator-Like Effector Nucleases)
تواريخ الأحداث: Date Created: 20160714 Date Completed: 20170606 Latest Revision: 20240104
رمز التحديث: 20240104
مُعرف محوري في PubMed: PMC5113113
DOI: 10.1038/mt.2016.148
PMID: 27406980
قاعدة البيانات: MEDLINE
الوصف
تدمد:1525-0024
DOI:10.1038/mt.2016.148