合成聚合物输送 RNA 的趋势。

IF 4.3 3区 材料科学 Q1 ENGINEERING, ELECTRICAL & ELECTRONIC ACS Applied Electronic Materials Pub Date : 2024-02-21 DOI:10.1002/jgm.3672
Josh J. Friesen, Anna K. Blakney
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引用次数: 0

摘要

核糖核酸(RNA)已成为基因治疗领域最有前途的治疗载体之一。核糖核酸有许多独特的类型,可用于疫苗接种、蛋白质替代疗法、自身免疫性疾病治疗、基因敲除和基因编辑等一系列应用。然而,RNA 会触发宿主免疫系统,容易被降解,而且不易自发进入细胞。因此,需要一种输送载体来促进 RNA 治疗药物的保护和被所需宿主细胞吸收。脂质纳米颗粒已成为唯一获得临床批准的基因有效载荷载体,包括 COVID-19 信使 RNA 疫苗。虽然脂质纳米粒子具有明显的优势,但也有缺点,包括强烈的免疫刺激、复杂的制造工艺和配方异质性。相比之下,合成聚合物是一类被广泛研究的基因递送载体,具有独特的优势,包括生物相容性、可调性、低致敏性、配方简单和易于改性。某些类别的聚合物可提高转染效率,降低对宿主免疫系统的刺激,因此更适合用于非疫苗相关的 RNA 药物应用。本综述旨在确定最有前途的几类合成聚合物,总结近期旨在将它们应用于临床的研究,并预测未来释放其全部潜力所需的步骤。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Trends in the synthetic polymer delivery of RNA

Ribonucleic acid (RNA) has emerged as one of the most promising therapeutic payloads in the field of gene therapy. There are many unique types of RNA that allow for a range of applications including vaccination, protein replacement therapy, autoimmune disease treatment, gene knockdown and gene editing. However, RNA triggers the host immune system, is vulnerable to degradation and has a low proclivity to enter cells spontaneously. Therefore, a delivery vehicle is required to facilitate the protection and uptake of RNA therapeutics into the desired host cells. Lipid nanoparticles have emerged as one of the only clinically approved vehicles for genetic payloads, including in the COVID-19 messenger RNA vaccines. While lipid nanoparticles have distinct advantages, they also have drawbacks, including strong immune stimulation, complex manufacturing and formulation heterogeneity. In contrast, synthetic polymers are a widely studied group of gene delivery vehicles and boast distinct advantages, including biocompatibility, tunability, inexpensiveness, simple formulation and ease of modification. Some classes of polymers enhance efficient transfection efficiency, and lead to lower stimulation of the host immune system, making them more viable candidates for non-vaccine-related applications of RNA medicines. This review aims to identify the most promising classes of synthetic polymers, summarize recent research aimed at moving them into the clinic and postulate the future steps required for unlocking their full potential.

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CiteScore
7.20
自引率
4.30%
发文量
567
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