Yu etal_ISCIENCE-D-23-06561: A Non-Viral DNA Delivery System Consisting of Multifunctional Chimaeric Peptide Fused with Zinc Finger Protein

Non-viral gene delivery systems have received sustained attention as a promising alternative to viral vectors for disease treatment and prevention in recent years. Numerous methods have been developed to enhance gene uptake and delivery in the cytoplasm, however, due to technical difficulties and delivery efficiency, these systems still face challenges in a range of biological applications, especially in vivo. To alleviate this challenge, we devised a novel system for gene delivery based on a recombinant protein eTAT-ZF9-NLS, which consisted of a multifunctional chimaeric peptide and a zinc finger protein with sequence-specific DNA-binding activity. High transfection efficiency was observed in several mammalian cells after intracellular delivery of plasmid containing ZF9-binding sites mediated by eTAT-ZF9-NLS. Our new approach provides a novel transfection strategy and the transfection efficiency was confirmed both in vitro and in vivo, making it a preferential transfection reagent for possible gene therapy.
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近年来，非病毒基因递送系统作为在疾病治疗与预防中可替代病毒载体的极具前景的方案，受到了持续关注。目前已开发出多种方法以提升基因在细胞质中的摄取与递送效率，但受限于技术难题与递送效能不足，此类系统在诸多生物学应用场景中仍面临挑战，尤其是在体内（in vivo）环境中。为缓解这一难题，我们开发了一种基于重组蛋白eTAT-ZF9-NLS的新型基因递送系统，该系统由多功能嵌合肽与具备序列特异性DNA结合活性的锌指蛋白构成。在经eTAT-ZF9-NLS介导的、携带有ZF9结合位点的质粒完成胞内递送后，多种哺乳动物细胞均展现出了较高的转染效率。本研究提出的新型策略为转染提供了全新方案，其转染效率已在体外（in vitro）与体内（in vivo）环境中得到验证，有望成为基因治疗领域极具应用潜力的转染试剂。
本数据集所含Excel文件均按对应图表进行编排，内含用于生成刊载于论文"ISCIENCE-D-23-06561"中各类图表的原始数据。