外泌体作为 mRNA 的递送载体:策略与治疗应用。

Exosome for mRNA delivery: strategies and therapeutic applications.

机构信息

Department of Orthopedics, Shenzhen Second People's Hospital, The First Affiliated Hospital of Shenzhen University, Shenzhen, 518035, China.

Department of Allied Health Sciences, The University of Agriculture, D.I.Khan, Pakistan.

出版信息

J Nanobiotechnology. 2024 Jul 4;22(1):395. doi: 10.1186/s12951-024-02634-x.

Abstract

Messenger RNA (mRNA) has emerged as a promising therapeutic molecule with numerous clinical applications in treating central nervous system disorders, tumors, COVID-19, and other diseases. mRNA therapies must be encapsulated into safe, stable, and effective delivery vehicles to preserve the cargo from degradation and prevent immunogenicity. Exosomes have gained growing attention in mRNA delivery because of their good biocompatibility, low immunogenicity, small size, unique capacity to traverse physiological barriers, and cell-specific tropism. Moreover, these exosomes can be engineered to utilize the natural carriers to target specific cells or tissues. This targeted approach will enhance the efficacy and reduce the side effects of mRNAs. However, difficulties such as a lack of consistent and reliable methods for exosome purification and the efficient encapsulation of large mRNAs into exosomes must be addressed. This article outlines current breakthroughs in cell-derived vesicle-mediated mRNA delivery and its biomedical applications.

摘要

信使 RNA(mRNA)作为一种有前途的治疗分子,在治疗中枢神经系统疾病、肿瘤、COVID-19 和其他疾病方面具有众多临床应用。为了保护货物不被降解并防止免疫原性,mRNA 疗法必须封装到安全、稳定和有效的递药载体中。由于 exosomes 具有良好的生物相容性、低免疫原性、小尺寸、独特的穿越生理屏障的能力和细胞特异性趋向性,因此在 mRNA 递送上越来越受到关注。此外,这些 exosomes 可以被工程化以利用天然载体来靶向特定的细胞或组织。这种靶向方法将提高 mRNAs 的疗效并降低其副作用。然而,必须解决一些困难,例如缺乏一致和可靠的 exosomes 纯化方法,以及将大 mRNAs 高效封装到 exosomes 中的方法。本文概述了细胞衍生囊泡介导的 mRNA 递药及其在生物医学中的应用的最新突破。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fbef/11225225/d638547c1a4f/12951_2024_2634_Fig1_HTML.jpg

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