دورية أكاديمية
Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.
| العنوان: | Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates. |
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| المؤلفون: | Romero Z; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Lomova A; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, Los Angeles, CA, USA., Said S; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Miggelbrink A; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Kuo CY; Division of Allergy & Immunology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA., Campo-Fernandez B; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Hoban MD; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Masiuk KE; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Clark DN; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Long J; Division of Allergy & Immunology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA., Sanchez JM; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Velez M; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Miyahira E; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Zhang R; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Brown D; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Wang X; Department of Medicine Statistics Core, University of California, Los Angeles, Los Angeles, CA, USA., Kurmangaliyev YZ; Department of Biological Chemistry, HHMI, University of California, Los Angeles, Los Angeles, CA, USA., Hollis RP; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA., Kohn DB; Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, Los Angeles, CA, USA; Department of Molecular and Medical Pharmacology, University of California, Los Angeles, Los Angeles, CA, USA; Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles, CA, USA. Electronic address: dkohn1@mednet.ucla.edu. |
| المصدر: | Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2019 Aug 07; Vol. 27 (8), pp. 1389-1406. Date of Electronic Publication: 2019 May 24. |
| نوع المنشور: | Journal Article; 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: | Gene Editing* , Mutation*, Anemia, Sickle Cell/*genetics , Hematopoietic Stem Cells/*metabolism , beta-Globins/*genetics, Anemia, Sickle Cell/metabolism ; Anemia, Sickle Cell/therapy ; CRISPR-Cas Systems ; Dependovirus ; Endonucleases/genetics ; Gene Expression ; Gene Targeting ; Genetic Therapy ; Genetic Vectors/genetics ; Humans ; Parvovirinae/genetics ; Tissue Donors ; Transduction, Genetic ; Zinc Finger Nucleases/genetics |
| مستخلص: | Site-specific correction of a point mutation causing a monogenic disease in autologous hematopoietic stem and progenitor cells (HSPCs) can be used as a treatment of inherited disorders of the blood cells. Sickle cell disease (SCD) is an ideal model to investigate the potential use of gene editing to transvert a single point mutation at the β-globin locus (HBB). We compared the activity of zinc-finger nucleases (ZFNs) and CRISPR/Cas9 for editing, and homologous donor templates delivered as single-stranded oligodeoxynucleotides (ssODNs), adeno-associated virus serotype 6 (AAV6), integrase-deficient lentiviral vectors (IDLVs), and adenovirus 5/35 serotype (Ad5/35) to transvert the base pair responsible for SCD in HBB in primary human CD34+ HSPCs. We found that the ZFNs and Cas9 directed similar frequencies of nuclease activity. In vitro, AAV6 led to the highest frequencies of homology-directed repair (HDR), but levels of base pair transversions were significantly reduced when analyzing cells in vivo in immunodeficient mouse xenografts, with similar frequencies achieved with either AAV6 or ssODNs. AAV6 also caused significant impairment of colony-forming progenitors and human cell engraftment. Gene correction in engrafting hematopoietic stem cells may be limited by the capacity of the cells to mediate HDR, suggesting additional manipulations may be needed for high-efficiency gene correction in HSPCs. (Copyright © 2019 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.) |
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| معلومات مُعتمدة: | R25 GM055052 United States GM NIGMS NIH HHS |
| فهرسة مساهمة: | Keywords: CRISPR/Cas9; adeno-associated virus 6; gene editing; hematopoietic stem cells; homologous recombination; non-homologous end joining; sickle cell disease; zinc finger nuclease |
| المشرفين على المادة: | 0 (beta-Globins) EC 3.1.- (Endonucleases) EC 3.1.- (Zinc Finger Nucleases) |
| SCR Organism: | Adeno-associated virus-1 |
| تواريخ الأحداث: | Date Created: 20190611 Date Completed: 20200529 Latest Revision: 20211204 |
| رمز التحديث: | 20221213 |
| مُعرف محوري في PubMed: | PMC6697408 |
| DOI: | 10.1016/j.ymthe.2019.05.014 |
| PMID: | 31178391 |
| قاعدة البيانات: | MEDLINE |
| تدمد: | 1525-0024 |
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| DOI: | 10.1016/j.ymthe.2019.05.014 |