دورية أكاديمية

CRISPR-Cas9 base editors and their current role in human therapeutics.

التفاصيل البيبلوغرافية
العنوان: CRISPR-Cas9 base editors and their current role in human therapeutics.
المؤلفون: Lahr WS; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA., Sipe CJ; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA., Skeate JG; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA., Webber BR; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA., Moriarity BS; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA. Electronic address: mori0164@umn.edu.
المصدر: Cytotherapy [Cytotherapy] 2023 Mar; Vol. 25 (3), pp. 270-276. Date of Electronic Publication: 2023 Jan 10.
نوع المنشور: Review; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
اللغة: English
بيانات الدورية: Publisher: Elsevier Country of Publication: England NLM ID: 100895309 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1477-2566 (Electronic) Linking ISSN: 14653249 NLM ISO Abbreviation: Cytotherapy Subsets: MEDLINE
أسماء مطبوعة: Publication: 2013- : London : Elsevier
Original Publication: Oxford, England : ISIS Medical Media, c1999-
مواضيع طبية MeSH: CRISPR-Cas Systems*/genetics , Gene Editing*, Humans ; Genetic Therapy ; DNA
مستخلص: Background: Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification.
Methods: This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes.
Results: CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction.
Conclusion: As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.
(Published by Elsevier Inc.)
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معلومات مُعتمدة: R01 AI161017 United States AI NIAID NIH HHS; T32 HL007062 United States HL NHLBI NIH HHS; R21 CA237789 United States CA NCI NIH HHS; P50 CA136393 United States CA NCI NIH HHS; R21 AI163731 United States AI NIAID NIH HHS; P01 CA254849 United States CA NCI NIH HHS; R01 AI146009 United States AI NIAID NIH HHS
فهرسة مساهمة: Keywords: base editor; cancer immunotherapy; gene therapy; hematopoietic stem cell; multiplex gene editing; sickle cell
المشرفين على المادة: 10191-18-1 (BES)
9007-49-2 (DNA)
تواريخ الأحداث: Date Created: 20230112 Date Completed: 20230207 Latest Revision: 20240617
رمز التحديث: 20240617
مُعرف محوري في PubMed: PMC10887149
DOI: 10.1016/j.jcyt.2022.11.013
PMID: 36635153
قاعدة البيانات: MEDLINE
الوصف
تدمد:1477-2566
DOI:10.1016/j.jcyt.2022.11.013