Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; These authors contributed equally.
Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Institute for Nanobiotechnology, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; These authors contributed equally.
Trends Biotechnol. 2019 Mar;37(3):281-293. doi: 10.1016/j.tibtech.2018.08.010. Epub 2018 Sep 29.
Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.
基因组编辑技术，如锌指核酸酶（ZFNs）、转录激活因子样效应物核酸酶（TALENs）和规律成簇间隔短回文重复（CRISPR）相关蛋白系统，已经彻底改变了生物研究。每种生物技术都包含一种 DNA 结合蛋白，该蛋白可以被编程以识别并启动双链断裂（DSBs），从而进行特定基因的修饰。这些技术有可能被用于治疗由异常基因表达引起的疾病。为了在治疗上取得成功，它们的功能取决于其安全有效地递送到细胞核内。这篇综述讨论了基因组编辑工具递送方面的挑战，并强调了最近在非病毒递送上的创新，这些创新有可能克服这些限制，推动基因组编辑向患者护理的转化。
