Bai Xingjian, Hong Jack F, Yu Shan, Hu David Y, Chen Amy Y, Rich Constance A, Shi Silk J, Xu Sandy Y, Croucher Daniel M, Müssar Kristofer J, Meng Daniel W, Chen Jane L, Lahn Bruce T
VectorBuilder, Chicago, IL 60609, United States.
Future Institute of Gene Delivery Research, Guangzhou, 510663, China.
Nucleic Acids Res. 2025 Jul 19;53(14). doi: 10.1093/nar/gkaf697.
Plasmids are indispensable in life sciences research and therapeutics development. Currently, most labs custom-build their plasmids. As yet, no systematic data on the quality of lab-made plasmids exist. Here, we report a broad survey of plasmids from hundreds of academic and industrial labs worldwide. We show that nearly half of them contained design and/or sequence errors. For transfer plasmids used in making AAV vectors, which are widely used in gene therapy, about 40% carried mutations in the inverted terminal repeat (ITR) regions due to their inherent instability, which is influenced by flanking GC content. We also list genes difficult to clone into plasmid or package into virus due to their toxicity. Our finding raises serious concerns over the trustworthiness of lab-made plasmids, which parallels the underappreciated mycoplasma contamination and misidentified mammalian cell lines reported previously, and highlights the need for community-wide standards to uphold the quality of this ubiquitous reagent in research and medicine. Accordingly, we propose the concept of good vector practice (GVP) that covers the proper design, construction, in-process QC, final QC, banking and management of plasmids in research and medicine to uphold their quality.
质粒在生命科学研究和治疗学发展中不可或缺。目前,大多数实验室都是定制构建自己的质粒。迄今为止,尚无关于实验室自制质粒质量的系统性数据。在此,我们报告了一项对来自全球数百个学术和工业实验室的质粒进行的广泛调查。我们发现,其中近一半含有设计和/或序列错误。在用于制造腺相关病毒(AAV)载体的转移质粒中,这种载体在基因治疗中被广泛使用,约40%由于其固有的不稳定性,在反向末端重复(ITR)区域携带突变,这种不稳定性受侧翼GC含量的影响。我们还列出了由于毒性而难以克隆到质粒中或包装到病毒中的基因。我们的发现引发了对实验室自制质粒可信度的严重担忧,这与之前未得到充分重视的支原体污染和错误鉴定的哺乳动物细胞系情况类似,并突出了在研究和医学中需要全行业标准来保证这种普遍使用的试剂的质量。因此,我们提出了良好载体操作规范(GVP)的概念,它涵盖了在研究和医学中质粒的合理设计、构建、过程质量控制、最终质量控制、储存和管理,以保证其质量。