دورية أكاديمية
أعرض في EDS

Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases.

التفاصيل البيبلوغرافية
العنوان: Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases.
المؤلفون: Bjurström CF; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Mojadidi M; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Phillips J; Howard Hughes Medical Institute, University of California, Los Angeles, Los Angeles, California, USA., Kuo C; Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA., Lai S; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Lill GR; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Cooper A; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Kaufman M; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Urbinati F; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Wang X; Department of General Internal Medicine and Health Services Research, University of California, Los Angeles, Los Angeles, California, USA., Hollis RP; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA., Kohn DB; Department of Microbiology, Immunology, & Molecular Genetics, University of California, Los Angeles, Los Angeles, California, USA; Department of Pediatrics, University of California, Los Angeles, Los Angeles, California, USA. Electronic address: dkohn@mednet.ucla.edu.
المصدر: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2016; Vol. 5, pp. e351.
نوع المنشور: Journal Article
اللغة: English
بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: Print Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE
أسماء مطبوعة: Publication: 2017- : Cambridge, MA : Cell Press
Original Publication: New York, NY : Nature Pub. Group
مستخلص: We examined the efficiency, specificity, and mutational signatures of zinc finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 systems designed to target the gene encoding the transcriptional repressor BCL11A, in human K562 cells and human CD34 + progenitor cells. ZFNs and TALENs were delivered as in vitro transcribed mRNA through electroporation; CRISPR/Cas9 was codelivered by Cas9 mRNA with plasmid-encoded guideRNA (gRNA) (pU6.g1) or in vitro transcribed gRNA (gR.1). Analyses of efficacy revealed that for these specific reagents and the delivery methods used, the ZFNs gave rise to more allelic disruption in the targeted locus compared to the TALENs and CRISPR/Cas9, which was associated with increased levels of fetal hemoglobin in erythroid cells produced in vitro from nuclease-treated CD34 + cells. Genome-wide analysis to evaluate the specificity of the nucleases revealed high specificity of this specific ZFN to the target site, while specific TALENs and CRISPRs evaluated showed off-target cleavage activity. ZFN gene-edited CD34 + cells had the capacity to engraft in NOD-Prkdc SCID -IL2Rγ null mice, while retaining multi-lineage potential, in contrast to TALEN gene-edited CD34 + cells. CRISPR engraftment levels mirrored the increased relative plasmid-mediated toxicity of pU6.g1/Cas9 in hematopoietic stem/progenitor cells (HSPCs), highlighting the value for the further improvements of CRISPR/Cas9 delivery in primary human HSPCs.
(Copyright © 2016 Official journal of the American Society of Gene & Cell Therapy. Published by Elsevier Inc. All rights reserved.)
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معلومات مُعتمدة: P01 HL073104 United States HL NHLBI NIH HHS; T32 GM007185 United States GM NIGMS NIH HHS
فهرسة مساهمة: Keywords: BCL11A; CRISPR/Cas9; TALENs; fetal hemoglobin; nonhomologous end joining; zinc finger nucleases
تواريخ الأحداث: Date Created: 20170130 Latest Revision: 20240922
رمز التحديث: 20240922
مُعرف محوري في PubMed: PMC5023398
DOI: 10.1038/mtna.2016.52
PMID: 28131278
قاعدة البيانات: MEDLINE
الوصف
تدمد:2162-2531
DOI:10.1038/mtna.2016.52