293变异体腺相关病毒生产能力差异遗传基础的生物信息学分析。

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Human gene therapy Pub Date : 2025-05-01 Epub Date: 2025-04-28 DOI:10.1089/hum.2025.002
Christopher R Herzog, Junping Zhang, Xiaomin Feng, Thao Thi Dang, Xiangping Yu, Jie Huang, Fang Fang, Hongyu Gao, Xuhong Yu, Yue Wang, Renzhi Han, Yulong Liu, Kenneth Cornetta, Weidong Xiao, Weihong Xu
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引用次数: 0

摘要

人胚胎肾293 (HEK 293)细胞是重组腺相关病毒(rAAV)的主要生产细胞系。然而,293个克隆的AAV载体产量差异很大。为了阐明这些差异的生物学基础,利用纳米孔技术对具有高产rAAV的293细胞贴壁克隆和悬浮克隆的全基因组进行了测序。所有293细胞衍生系在腺病毒整合位点的拷贝数增加了两倍,表明存在基因组复制事件。令我们惊讶的是,这两个高产克隆,尽管是分开培育的,但与其他常用的293克隆相比,在生物学上却紧密地聚集在一起。它们的基因组包含一个类似的腺病毒基因整合区,这可能导致促进AAV复制和包装的蛋白质的高表达。因此,腺病毒整合位点的基因组重复可能是影响AAV产量的关键因素。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Bioinformatic Analysis of the Genetic Basis of Differential Adeno-Associated Virus Production Capability of 293 Variants.

Human embryonic kidney 293 (HEK 293) cells are the main producer cell line for recombinant adeno-associated virus (rAAV) production. However, AAV vector yields among 293 clones vary considerably. To elucidate the biological basis for these differences, whole genomes of an adherent and a suspension 293 cell clone with high-yield rAAV were sequenced using nanopore technology. All 293 cell derivative lines showed a twofold copy number gain at the adenoviral integration site across, suggesting a genome duplication event. To our surprise, the two high-producer clones, despite having been separately developed, are biologically closely grouped together as compared to other commonly used 293 clones. Their genomes contain a similar adenoviral gene integration region, which likely leads to high expression of proteins that facilitate AAV replication and packaging. Thus, genome duplication in the adenovirus integration locus may be a key factor affecting AAV production yield.

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来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
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