Kim Dongyoon, Le Quoc-Viet, Wu Yina, Park Jinwon, Oh Yu-Kyoung
College of Pharmacy and Research Institute of Pharmaceutical Sciences, Seoul National University, Seoul 08826, Korea.
Pharmaceutics. 2020 Dec 18;12(12):1233. doi: 10.3390/pharmaceutics12121233.
Genome-editing technology has emerged as a potential tool for treating incurable diseases for which few therapeutic modalities are available. In particular, discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system together with the design of single-guide RNAs (sgRNAs) has sparked medical applications of genome editing. Despite the great promise of the CRISPR/Cas system, its clinical application is limited, in large part, by the lack of adequate delivery technology. To overcome this limitation, researchers have investigated various systems, including viral and nonviral vectors, for delivery of CRISPR/Cas and sgRNA into cells. Among nonviral delivery systems that have been studied are nanovesicles based on lipids, polymers, peptides, and extracellular vesicles. These nanovesicles have been designed to increase the delivery of CRISPR/Cas and sgRNA through endosome escape or using various stimuli such as light, pH, and environmental features. This review covers the latest research trends in nonviral, nanovesicle-based delivery systems that are being applied to genome-editing technology and suggests directions for future progress.
基因组编辑技术已成为一种潜在工具，用于治疗几乎没有可用治疗方式的不治之症。特别是，成簇规律间隔短回文重复序列（CRISPR）/Cas系统的发现以及单向导RNA（sgRNA）的设计引发了基因组编辑的医学应用。尽管CRISPR/Cas系统前景广阔，但其临床应用在很大程度上受到缺乏足够递送技术的限制。为克服这一限制，研究人员研究了各种系统，包括病毒和非病毒载体，用于将CRISPR/Cas和sgRNA递送至细胞。在已研究的非病毒递送系统中，有基于脂质、聚合物、肽和细胞外囊泡的纳米囊泡。这些纳米囊泡旨在通过内体逃逸或利用光、pH值和环境特征等各种刺激来增加CRISPR/Cas和sgRNA的递送。本综述涵盖了应用于基因组编辑技术的基于纳米囊泡的非病毒递送系统的最新研究趋势，并提出了未来进展的方向。
