Genetics and Genomic Medicine, Great Ormond Street Institute of Child Health, University College London, London, UK.
Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Br J Pharmacol. 2021 Jun;178(12):2375-2392. doi: 10.1111/bph.15432. Epub 2021 May 3.
Exosomes are a subset of extracellular vesicles essential for cell-cell communication in health and disease with the ability to transport nucleic acids, functional proteins and other metabolites. Their clinical use as diagnostic biomarkers and therapeutic carriers has become a major field of research over recent years, generating rapidly expanding scientific interest and financial investment. Their reduced immunogenicity compared to liposomes or viral vectors and their ability to cross major physiological barriers like the blood-brain barrier make them an appealing and innovative option as biomarkers and therapeutic agents. Here, we review the latest clinical developments of exosome biotechnology for diagnostic and therapeutic purposes, including the most recent COVID-19-related exosome-based clinical trials. We present current exosome engineering strategies for optimal clinical safety and efficacy, and assess the technology developed for good manufacturing practice compliant scaling up and storage approaches along with their limitations in pharmaceutical industry.
外泌体是细胞外囊泡的一个子集,对于健康和疾病中的细胞间通讯至关重要,具有运输核酸、功能蛋白和其他代谢物的能力。近年来,它们作为诊断生物标志物和治疗载体的临床应用已成为一个主要的研究领域,引起了迅速增长的科学兴趣和资金投入。与脂质体或病毒载体相比,外泌体的免疫原性较低,并且能够穿过血脑屏障等主要生理屏障,这使它们成为作为生物标志物和治疗剂的一种有吸引力和创新的选择。在这里,我们综述了外泌体生物技术在诊断和治疗方面的最新临床进展,包括最近与 COVID-19 相关的基于外泌体的临床试验。我们介绍了目前用于优化临床安全性和疗效的外泌体工程策略,并评估了为符合良好生产规范的放大和储存方法而开发的技术,以及它们在制药行业中的局限性。