Target-seq:检测基因组整合位点、DNA易位和脱靶事件的单一工作流程。

IF 2.2 4区 工程技术 Q3 BIOCHEMICAL RESEARCH METHODS
Pei-Zhong Tang, Bo Ding, Christopher Reyes, David Papp, Jason Potter
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引用次数: 0

摘要

设计供体DNA通过病毒或非病毒系统递送到宿主基因组中的靶位点是基因治疗的关键步骤。腺相关病毒和慢病毒由于其高传递和编辑效率,是体内和体外传递治疗基因的主要载体。非病毒编辑工具,如CRISPR/Cas9,在基因修饰方面正受到越来越多的关注。然而,也存在安全问题;例如,脱靶效应和DNA重排导致的肿瘤发生。检测和表征宿主基因组中靶向和脱靶基因组修饰后编辑的分析工具对于评估基因治疗的成功和安全性至关重要。我们结合不同的分析工具开发了Target-seq来检测基因组整合位点、DNA易位和脱靶事件。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Target-seq: single workflow for detection of genome integration site, DNA translocation and off-target events.

Designed donor DNA delivery through viral or nonviral systems to target loci in the host genome is a critical step for gene therapy. Adeno-associated virus and lentivirus are leading vehicles for in vivo and ex vivo delivery of therapeutic genes due to their high delivery and editing efficiency. Nonviral editing tools, such as CRISPR/Cas9, are getting more attention for gene modification. However, there are safety concerns; for example, tumorigenesis due to off-target effects and DNA rearrangement. Analysis tools to detect and characterize on-target and off-target genome modification post editing in the host genome are pivotal for evaluating the success and safety of gene therapy. We developed Target-seq combined with different analysis tools to detect the genome integration site, DNA translocation and off-target events.

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来源期刊
BioTechniques
BioTechniques 工程技术-生化研究方法
CiteScore
4.40
自引率
0.00%
发文量
68
审稿时长
3.3 months
期刊介绍: BioTechniques is a peer-reviewed, open-access journal dedicated to publishing original laboratory methods, related technical and software tools, and methods-oriented review articles that are of broad interest to professional life scientists, as well as to scientists from other disciplines (e.g., chemistry, physics, computer science, plant and agricultural science and climate science) interested in life science applications for their technologies. Since 1983, BioTechniques has been a leading peer-reviewed journal for methods-related research. The journal considers: Reports describing innovative new methods, platforms and software, substantive modifications to existing methods, or innovative applications of existing methods, techniques & tools to new models or scientific questions Descriptions of technical tools that facilitate the design or performance of experiments or data analysis, such as software and simple laboratory devices Surveys of technical approaches related to broad fields of research Reviews discussing advancements in techniques and methods related to broad fields of research Letters to the Editor and Expert Opinions highlighting interesting observations or cautionary tales concerning experimental design, methodology or analysis.
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