RNA 疗法的开发和技术。

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Huong Lan Vuong, Chu Thanh Lan, Hien Thi Thu Le
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引用次数: 0

摘要

siRNA 自 20 多年前被发现以来,其在体外和体内基因沉默模型中的潜力已得到认可。对 RNA 的新一代、分子机制、靶点附着和纯化等方面的多项研究支持了 RNA 疗法的发展,使其应用领域更加广泛。RNA 疗法发展迅速,各种平台为个性化医疗和罕见病治疗标准做出了贡献。因此,了解 RNA 疗法的发展和技术成为新药研发的关键点。在此,本综述的主要目的是对基于 RNA 的治疗方法的六个治疗类别进行概述,包括 RNA 靶向治疗、蛋白质靶向治疗、细胞重编程和组织工程、基于 RNA 的蛋白质替代治疗、基于 RNA 的基因组编辑以及基于非编码 RNA 和编码 RNA 的 RNA 免疫疗法。此外,我们还概述了设计新一代 RNA 技术的病毒和非病毒 RNA 策略。我们还讨论了使用 RNA 疗法的优势和挑战,以及各种 RNA 递送方法。因此,本综述旨在为这一领域的研究人员提供最新的 RNA 疗法在对抗罕见或难治疾病方面的参考证据。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The development and technologies of RNA therapeutics.

Since it was discovered for over 20 years ago, the potentiality of siRNAs in gene silencing in vitro and in vivo models has been recognized. Several studies in the new generation, molecular mechanisms, target attachment, and purification of RNA have supported the development of RNA therapeutics for a variety of applications. RNA therapeutics are growing rapidly with various platforms contributing to the standard of personalized medicine and rare disease treatment. Therefore, understanding the development and technologies of RNA therapeutics becomes a crucial point for new drug generation. Here, the primary purpose of this review is to provide a general view of six therapeutic categories that make up RNA-based therapeutic approaches, including RNA-target therapeutics, protein-targeted therapeutics, cellular reprogramming and tissues engineering, RNA-based protein replacement therapeutics, RNA-based genome editing, and RNA-based immunotherapies based on non-coding RNAs and coding RNA. Furthermore, we present an overview of the RNA strategies regarding viral approaches and nonviral approaches in designing a new generation of RNA technologies. The advantages and challenges of using RNA therapeutics are also discussed along with various approaches for RNA delivery. Therefore, this review is designed to provide updated reference evidence of RNA therapeutics in the battle against rare or difficult-to-treat diseases for researchers in this field.

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来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
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