Current and prospective strategies for advancing the targeted delivery of CRISPR/Cas system via extracellular vesicles

• Published in: Journal of nanobiotechnology Vol. 21; no. 1; p. 184
• Main Authors: Huang, Xiaowen, Li, Aifang, Xu, Peng, Yu, Yangfan, Li, Shuxuan, Hu, Lina, Feng, Shuying
• Format: Journal Article
• Language: English
• Published: England BioMed Central Ltd 08.06.2023; BioMed Central; BMC
• Subjects: Biocompatibility; CRISPR; CRISPR/Cas system; Current efficiency; Efficiency; Extracellular vesicle; Extracellular vesicles; Gene editing; Gene therapy; Gene transfer; Genetic modification; Genome editing; Genomes; Health aspects; Immunogenicity; Molecular targeted therapy; Nanocarrier; Nanotechnology; Patient outcomes; Physiology; Plasmids; Proteins; Review; Targeted delivery; Toxicity; Vesicles; China; CRISPR/Cas system; Nanocarrier; Extracellular vesicle; Targeted delivery; Gene editing
• ISSN: 1477-3155; 1477-3155
• DOI: 10.1186/s12951-023-01952-w

Extracellular vesicles (EVs) have emerged as a promising platform for gene delivery owing to their natural properties and phenomenal functions, being able to circumvent the significant challenges associated with toxicity, problematic biocompatibility, and immunogenicity of the standard approaches. These features are of particularly interest for targeted delivery of the emerging clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated (Cas) systems. However, the current efficiency of EV-meditated transport of CRISPR/Cas components remains insufficient due to numerous exogenous and endogenous barriers. Here, we comprehensively reviewed the current status of EV-based CRISPR/Cas delivery systems. In particular, we explored various strategies and methodologies available to potentially improve the loading capacity, safety, stability, targeting, and tracking for EV-based CRISPR/Cas system delivery. Additionally, we hypothesise the future avenues for the development of EV-based delivery systems that could pave the way for novel clinically valuable gene delivery approaches, and may potentially bridge the gap between gene editing technologies and the laboratory/clinical application of gene therapies.