BrisSynBio Bristol Synthetic Biology Centre, Biomedical Sciences, School of Biochemistry, 1 Tankard's Close, University of Bristol, Bristol BS8 1TD, UK.
The Rolf Luft Research Center for Diabetes and Endocrinology, Karolinska Institutet, SE-171 76 Stockholm, Sweden.
Nucleic Acids Res. 2022 Jul 22;50(13):7783-7799. doi: 10.1093/nar/gkac587.
CRISPR-based precise gene-editing requires simultaneous delivery of multiple components into living cells, rapidly exceeding the cargo capacity of traditional viral vector systems. This challenge represents a major roadblock to genome engineering applications. Here we exploit the unmatched heterologous DNA cargo capacity of baculovirus to resolve this bottleneck in human cells. By encoding Cas9, sgRNA and Donor DNAs on a single, rapidly assembled baculoviral vector, we achieve with up to 30% efficacy whole-exon replacement in the intronic β-actin (ACTB) locus, including site-specific docking of very large DNA payloads. We use our approach to rescue wild-type podocin expression in steroid-resistant nephrotic syndrome (SRNS) patient derived podocytes. We demonstrate single baculovirus vectored delivery of single and multiplexed prime-editing toolkits, achieving up to 100% cleavage-free DNA search-and-replace interventions without detectable indels. Taken together, we provide a versatile delivery platform for single base to multi-gene level genome interventions, addressing the currently unmet need for a powerful delivery system accommodating current and future CRISPR technologies without the burden of limited cargo capacity.
基于 CRISPR 的精确基因编辑需要将多个组件同时递送到活细胞中,这迅速超过了传统病毒载体系统的载物能力。这一挑战代表了基因组工程应用的主要障碍。在这里,我们利用杆状病毒无与伦比的异源 DNA 载物能力来解决人类细胞中的这一瓶颈问题。通过在单个快速组装的杆状病毒载体上编码 Cas9、sgRNA 和供体 DNA,我们在β-肌动蛋白 (ACTB) 基因的内含子中实现了高达 30%的有效全外显子替换,包括非常大的 DNA 有效载荷的特异性对接。我们使用我们的方法来拯救类固醇耐药性肾病综合征 (SRNS) 患者来源的足细胞中野生型足细胞蛋白的表达。我们证明了单杆状病毒载体可递送单链和多链的先导编辑工具包,实现了高达 100%无切割的 DNA 搜索和替换干预,而没有可检测的插入缺失。总之,我们提供了一个多功能的递送平台,用于单碱基到多基因水平的基因组干预,解决了当前对强大的递送系统的需求,该系统可以适应当前和未来的 CRISPR 技术,而不会受到载物能力有限的负担。