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本文引用的文献

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Cas13d: A New Molecular Scissor for Transcriptome Engineering.Cas13d:用于转录组工程的新型分子剪刀
Front Cell Dev Biol. 2022 Mar 31;10:866800. doi: 10.3389/fcell.2022.866800. eCollection 2022.
2
Protein Splicing of Inteins: A Powerful Tool in Synthetic Biology.内含肽的蛋白质剪接:合成生物学中的强大工具。
Front Bioeng Biotechnol. 2022 Feb 21;10:810180. doi: 10.3389/fbioe.2022.810180. eCollection 2022.
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The Role of Recombinant AAV in Precise Genome Editing.重组腺相关病毒在精确基因组编辑中的作用。
Front Genome Ed. 2022 Jan 13;3:799722. doi: 10.3389/fgeed.2021.799722. eCollection 2021.
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Basic Principles and Clinical Applications of CRISPR-Based Genome Editing.基于 CRISPR 的基因组编辑的基本原理和临床应用。
Yonsei Med J. 2022 Feb;63(2):105-113. doi: 10.3349/ymj.2022.63.2.105.
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Non-Integrating Lentiviral Vectors in Clinical Applications: A Glance Through.临床应用中的非整合型慢病毒载体:概述
Biomedicines. 2022 Jan 5;10(1):107. doi: 10.3390/biomedicines10010107.
6
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.Cas9 特异性免疫反应会损害多种退行性犬模型中的局部和全身 AAV CRISPR 治疗。
Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7.
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Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo.通过体内同源定向修复实现精确 Cas9 基因组编辑的自失活、一体化 AAV 载体。
Nat Commun. 2021 Nov 1;12(1):6267. doi: 10.1038/s41467-021-26518-y.
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Using Synthetically Engineered Guide RNAs to Enhance CRISPR Genome Editing Systems in Mammalian Cells.利用合成工程化引导RNA增强哺乳动物细胞中的CRISPR基因组编辑系统。
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COVID-19 Vaccines: Adenoviral Vectors.新型冠状病毒肺炎(COVID-19)疫苗:腺病毒载体。
Annu Rev Med. 2022 Jan 27;73:41-54. doi: 10.1146/annurev-med-012621-102252. Epub 2021 Oct 5.
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delivery of CRISPR-Cas9 therapeutics: Progress and challenges.CRISPR-Cas9疗法的递送:进展与挑战。
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用于递送CRISPR组件的病毒载体:进展与挑战

Viral Vectors for the Delivery of CRISPR Components: Advances and Challenges.

作者信息

Asmamaw Mengstie Misganaw

机构信息

Department of Biochemistry, College of Medicine and Health Sciences, Debre Tabor University, Debre Tabor, Ethiopia.

出版信息

Front Bioeng Biotechnol. 2022 May 12;10:895713. doi: 10.3389/fbioe.2022.895713. eCollection 2022.

DOI:10.3389/fbioe.2022.895713
PMID:35646852
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9133430/
Abstract

The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) and its accompanying protein (Cas9) are now the most effective, efficient, and precise genome editing techniques. Two essential components of the CRISPR/Cas9 system are guide RNA (gRNA) and CRISPR-associated (Cas9) proteins. Choosing and implementing safe and effective delivery systems in the therapeutic application of CRISPR/Cas9 has proven to be a significant problem. For CRISPR/Cas9 delivery, viral vectors are the natural specialists. Due to their higher delivery effectiveness than other delivery methods, vectors such as adenoviral vectors (AdVs), adeno-associated viruses (AAVs), and lentivirus vectors (LVs) are now commonly employed as delivery methods. This review thoroughly examined recent achievements in using a variety of viral vectors as a means of CRISPR/Cas9 delivery, as well as the benefits and limitations of each viral vector. Future thoughts for overcoming the current restrictions and adapting the technology are also discussed.

摘要

成簇规律间隔短回文重复序列(CRISPR)及其相关蛋白(Cas9)是目前最有效、高效且精确的基因组编辑技术。CRISPR/Cas9系统的两个关键组件是向导RNA(gRNA)和CRISPR相关(Cas9)蛋白。在CRISPR/Cas9的治疗应用中选择并实施安全有效的递送系统已被证明是一个重大问题。对于CRISPR/Cas9递送而言,病毒载体是天生的行家。由于其递送效率高于其他递送方法,腺病毒载体(AdV)、腺相关病毒(AAV)和慢病毒载体(LV)等载体目前通常被用作递送手段。本综述全面研究了使用多种病毒载体作为CRISPR/Cas9递送手段的最新成果,以及每种病毒载体的优缺点。还讨论了克服当前限制并改进该技术的未来思路。