Inhaled non-viral delivery systems for RNA therapeutics

IF 14.7 1区医学 Q1 PHARMACOLOGY & PHARMACY

Acta Pharmaceutica Sinica. B Pub Date : 2025-05-01 DOI:10.1016/j.apsb.2025.03.033

Cheng Huang , Hongjian Li , Xing Duan , Peidong Zhang , Shaolong Qi , Jianshi Du , Xiangrong Song , Aiping Tong , Guocan Yu

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引用次数: 0

Abstract

RNA-based gene therapy has been widely used for various diseases, and extensive studies have proved that suitable delivery routes greatly help the development of RNA therapeutics. Identifying a safe and effective delivery system is key to realizing RNA therapeutics’ clinical translation. Inhalation is a non-invasive pulmonary delivery modality that can enhance the retention of therapeutic agents in the lungs with negligible toxicity, thereby improving patient compliance. Inhaled RNA therapeutics are increasingly becoming an area of focus for researchers; however, only several clinical trials have explored inhaled delivery of RNA for pulmonary diseases. This review presents an overview of recent advances in inhaled delivery systems for RNA therapeutics, including viral and nonviral systems, highlighting state of the art regarding inhalation in the messenger RNA (mRNA) field. We also summarize the applications of mRNA inhalants in infectious and other lung diseases. Simultaneously, the research progresses on small interfering RNAs (siRNAs), antisense oligonucleotides (ASOs), and different types of RNA are also discussed to provide new strategies for developing RNA inhalation therapy. Finally, we clarify the challenges inhaled RNA-based therapeutics face before their widespread adoption and provide insights to help advance this exciting field to the bedside.

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用于RNA治疗的吸入非病毒递送系统

基于RNA的基因治疗已广泛应用于各种疾病，大量研究证明，合适的递送途径极大地促进了RNA疗法的发展。确定一种安全有效的递送系统是实现RNA疗法临床转化的关键。吸入是一种无创的肺部给药方式，可以增强治疗药物在肺部的潴留，毒性可以忽略不计，从而提高患者的依从性。吸入RNA疗法正日益成为研究人员关注的一个领域；然而，只有几项临床试验探索了吸入递送RNA治疗肺部疾病。本文综述了RNA治疗吸入递送系统的最新进展，包括病毒和非病毒系统，重点介绍了信使RNA （mRNA）吸入领域的最新进展。我们还总结了mRNA吸入剂在感染性和其他肺部疾病中的应用。同时，对小干扰RNA （sirna）、反义寡核苷酸（ASOs）和不同类型RNA的研究进展进行了讨论，以期为RNA吸入疗法的发展提供新的策略。最后，我们澄清了吸入rna疗法在广泛应用之前面临的挑战，并提供了有助于将这一令人兴奋的领域推进到床边的见解。

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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来源期刊

Acta Pharmaceutica Sinica. B Pharmacology, Toxicology and Pharmaceutics-General Pharmacology, Toxicology and Pharmaceutics

CiteScore

22.40

自引率

5.50%

发文量

1051

审稿时长

19 weeks

期刊介绍： The Journal of the Institute of Materia Medica, Chinese Academy of Medical Sciences, and the Chinese Pharmaceutical Association oversees the peer review process for Acta Pharmaceutica Sinica. B (APSB). Published monthly in English, APSB is dedicated to disseminating significant original research articles, rapid communications, and high-quality reviews that highlight recent advances across various pharmaceutical sciences domains. These encompass pharmacology, pharmaceutics, medicinal chemistry, natural products, pharmacognosy, pharmaceutical analysis, and pharmacokinetics. A part of the Acta Pharmaceutica Sinica series, established in 1953 and indexed in prominent databases like Chemical Abstracts, Index Medicus, SciFinder Scholar, Biological Abstracts, International Pharmaceutical Abstracts, Cambridge Scientific Abstracts, and Current Bibliography on Science and Technology, APSB is sponsored by the Institute of Materia Medica, Chinese Academy of Medical Sciences, and the Chinese Pharmaceutical Association. Its production and hosting are facilitated by Elsevier B.V. This collaborative effort ensures APSB's commitment to delivering valuable contributions to the pharmaceutical sciences community.