细胞外囊泡与脂质纳米颗粒在递送核酸方面的比较

IF 15.2 1区医学 Q1 PHARMACOLOGY & PHARMACY

Advanced drug delivery reviews Pub Date : 2024-10-28 DOI:10.1016/j.addr.2024.115461

Johannes Bader, Finn Brigger, Jean-Christophe Leroux

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引用次数: 0

摘要

细胞外囊泡（EVs）在细胞间信号转导中运输核酸（NA）和基于蛋白质的货物方面发挥着天然作用，因此越来越多的研究将其用于递送核酸（NA）治疗药物。在过去的几十年里，人们开发了脂质纳米颗粒（LNPs）作为核酸载体，并最终批准了几种上市配方，如 patisiran/Onpattro® 和 mRNA-1273/BNT162 COVID-19 疫苗。LNPs 的成功促使人们努力开发针对肝外器官的创新技术，并提供新的治疗方式，如体内基因编辑工具。随着这两个领域的最新进展，本综述旨在从EV的生物发生到LNP的结构特性，全面概述EV和基于LNP的NA递送系统的基本特征。它从药物配制和递送的角度探讨了利用这些纳米载体所遇到的主要挑战。此外，还比较了生物分布特征、体外和体内转染结果以及它们在临床试验中的状况。总之，本综述为基于 EV 和 LNP 的 NA 给药系统提供了有前途的研究途径和潜在的死胡同。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Extracellular vesicles versus lipid nanoparticles for the delivery of nucleic acids

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Extracellular vesicles versus lipid nanoparticles for the delivery of nucleic acids

Extracellular vesicles (EVs) are increasingly investigated for delivering nucleic acid (NA) therapeutics, leveraging their natural role in transporting NA and protein-based cargo in cell-to-cell signaling. Their synthetic counterparts, lipid nanoparticles (LNPs), have been developed over the past decades as NA carriers, culminating in the approval of several marketed formulations such as patisiran/Onpattro® and the mRNA-1273/BNT162 COVID-19 vaccines. The success of LNPs has sparked efforts to develop innovative technologies to target extrahepatic organs, and to deliver novel therapeutic modalities, such as tools for in vivo gene editing. Fueled by the recent advancements in both fields, this review aims to provide a comprehensive overview of the basic characteristics of EV and LNP-based NA delivery systems, from EV biogenesis to structural properties of LNPs. It addresses the primary challenges encountered in utilizing these nanocarriers from a drug formulation and delivery perspective. Additionally, biodistribution profiles, in vitro and in vivo transfection outcomes, as well as their status in clinical trials are compared. Overall, this review provides insights into promising research avenues and potential dead ends for EV and LNP-based NA delivery systems.

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来源期刊

Advanced drug delivery reviews 医学-药学

CiteScore

28.10

自引率

5.00%

发文量

294

审稿时长

15.1 weeks

期刊介绍： The aim of the Journal is to provide a forum for the critical analysis of advanced drug and gene delivery systems and their applications in human and veterinary medicine. The Journal has a broad scope, covering the key issues for effective drug and gene delivery, from administration to site-specific delivery. In general, the Journal publishes review articles in a Theme Issue format. Each Theme Issue provides a comprehensive and critical examination of current and emerging research on the design and development of advanced drug and gene delivery systems and their application to experimental and clinical therapeutics. The goal is to illustrate the pivotal role of a multidisciplinary approach to modern drug delivery, encompassing the application of sound biological and physicochemical principles to the engineering of drug delivery systems to meet the therapeutic need at hand. Importantly the Editorial Team of ADDR asks that the authors effectively window the extensive volume of literature, pick the important contributions and explain their importance, produce a forward looking identification of the challenges facing the field and produce a Conclusions section with expert recommendations to address the issues.