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利用PASTE在哺乳动物细胞中进行精确的千碱基规模基因组插入。

Precise kilobase-scale genomic insertions in mammalian cells using PASTE.

作者信息

Fell Christopher W, Schmitt-Ulms Cian, Tagliaferri Dario V, Gootenberg Jonathan S, Abudayyeh Omar O

机构信息

Harvard Medical School, Harvard University, Boston, MA, USA.

Division of Engineering in Medicine, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Cambridge, MA, USA.

出版信息

Nat Protoc. 2024 Dec 15. doi: 10.1038/s41596-024-01090-z.

Abstract

Programmable gene integration technologies are an emerging modality with exciting applications in both basic research and therapeutic development. Programmable addition via site-specific targeting elements (PASTE) is a programmable gene integration approach for precise and efficient programmable integration of large DNA sequences into the genome. PASTE offers improved editing efficiency, purity and programmability compared with previous methods for long insertions into the mammalian genome. By combining the specificity and cargo size capabilities of site-specific integrases with the programmability of prime editing, PASTE can precisely insert cargoes of at least 36 kb with efficiencies of up to 60%. Here we outline best practices for design, execution and analysis of PASTE experiments, with protocols for integration of EGFP at the human NOLC1 and ACTB genomic loci and for readout by next generation sequencing and droplet digital PCR. We provide guidelines for designing and optimizing a custom PASTE experiment for integration of desired payloads at alternative genomic loci, as well as example applications for in-frame protein tagging and multiplexed insertions. To facilitate experimental setup, we include the necessary sequences and plasmids for the delivery of PASTE components to cells via plasmid transfection or in vitro transcribed RNA. Most experiments in this protocol can be performed in as little as 2 weeks, allowing for precise and versatile programmable gene insertion.

摘要

可编程基因整合技术是一种新兴的技术形式,在基础研究和治疗开发中都有令人兴奋的应用。通过位点特异性靶向元件进行可编程添加(PASTE)是一种可编程基因整合方法,用于将大的DNA序列精确、高效地可编程整合到基因组中。与以前用于向哺乳动物基因组中进行长片段插入的方法相比,PASTE具有更高的编辑效率、纯度和可编程性。通过将位点特异性整合酶的特异性和载体大小能力与碱基编辑的可编程性相结合,PASTE可以精确插入至少36 kb的载体,效率高达60%。在这里,我们概述了PASTE实验设计、执行和分析的最佳实践,以及在人类NOLC1和ACTB基因组位点整合EGFP并通过下一代测序和液滴数字PCR进行读出的方案。我们提供了在替代基因组位点设计和优化定制PASTE实验以整合所需载荷的指导方针,以及框内蛋白质标签和多重插入的示例应用。为便于实验设置,我们提供了通过质粒转染或体外转录RNA将PASTE组件递送至细胞所需的序列和质粒。本方案中的大多数实验可在短短2周内完成,从而实现精确且通用的可编程基因插入。

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