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Inhaled non-viral delivery systems for RNA therapeutics

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收录情况： ◇ 统计源期刊 ◇ CSCD-C ◇ 卓越：领军期刊

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机构： [1]Key Laboratory of Bioorganic Phosphorus Chemistry & Chemical Biology, Department of Chemistry, Tsinghua University, Beijing 100084, China. [2]State Key Laboratory of Biotherapy and Cancer Center, Research Unit of Gene and Immunotherapy, Chinese Academy of Medical Sciences, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China. [3]Institute for Immunology, School of Basic Medical Sciences, Tsinghua University, Beijing 100084, China. [4]Vascular Surgery Center, the Third Hospital of Jilin University, Changchun 130031, China.

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关键词： Inhalation therapy mRNA Non-viral vectors Lipid nanoparticle Pulmonary diseases

摘要：

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.© 2025 The Authors.

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出版当年[2025]版：

大类 | 1 区

医学

小类 | 1 区药学

最新[2025]版：

大类 | 1 区

医学

小类 | 1 区药学

第一作者：

第一作者机构： [1]Key Laboratory of Bioorganic Phosphorus Chemistry & Chemical Biology, Department of Chemistry, Tsinghua University, Beijing 100084, China. [2]State Key Laboratory of Biotherapy and Cancer Center, Research Unit of Gene and Immunotherapy, Chinese Academy of Medical Sciences, Collaborative Innovation Center of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China.

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通讯作者：

通讯机构： [1]Key Laboratory of Bioorganic Phosphorus Chemistry & Chemical Biology, Department of Chemistry, Tsinghua University, Beijing 100084, China. [3]Institute for Immunology, School of Basic Medical Sciences, Tsinghua University, Beijing 100084, China.

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