Therapeutic application of extracellular vesicles in human diseases.

IF 12 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Molecular Therapy Pub Date : 2025-05-07 Epub Date: 2025-04-03 DOI:10.1016/j.ymthe.2025.04.002
Allancer Nunes, Tianpeng Zhang, Xiaodong Mu, Paul D Robbins
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Abstract

Extracellular vesicles (EVs) are membrane vesicles released or secreted from almost all cell types. EVs are derived from multivesicular bodies or from the plasma membrane and contain a subset of proteins, lipids, and nucleic acids (e.g., DNA, RNA, and microRNA [miRNA]) derived from the parent cell. EVs play important roles in intercellular communication by efficiently transferring the content between cells both locally and systemically. Given their natural ability to transfer cargo to cells, sometimes in a targeted manner, and their apparent lack of immunogenicity, EVs are being engineered for delivery of therapeutic RNAs, DNAs, miRNAs, viral particles, drugs, and even proteins. In addition, many of the therapeutic effects of stem cell treatments are mediated by stem cell-derived EVs, which are safer and potentially more effective than the parental stem cells. Here we provide an overview of the use of EVs for delivery of different therapeutic nucleic acids, viruses, and drugs, as well as the use of therapeutic stem cell-derived EVs.

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细胞外囊泡在人类疾病中的治疗应用。
细胞外囊泡(EVs)是几乎所有细胞类型释放或分泌的膜囊泡。ev来源于多泡体或质膜,含有来自亲本细胞的蛋白质、脂质和核酸(如DNA、RNA和miRNA)。电动汽车在细胞间通讯中发挥重要作用,有效地在细胞间传递局部和系统的内容物。考虑到它们将货物运送到细胞的天然能力(有时是以靶向的方式),以及它们明显缺乏免疫原性,电动汽车正被设计用于运送治疗性rna、dna、mirna、病毒颗粒、药物甚至蛋白质。此外,干细胞治疗的许多治疗效果是由干细胞衍生的ev介导的,它比亲本干细胞更安全,也可能更有效。在这里,我们概述了使用ev来递送不同的治疗性核酸、病毒和药物,以及使用治疗性干细胞衍生的ev。
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来源期刊
Molecular Therapy
Molecular Therapy 医学-生物工程与应用微生物
CiteScore
19.20
自引率
3.20%
发文量
357
审稿时长
3 months
期刊介绍: Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.
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Innovative approach in malaria research: Harnessing CRISPR-Cas9 for antimalarial drug-resistance studies in Africa. Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies. Igniting CAR-NKT cells with IL-18. PKC activators improve the function of biogenesis-deficient CFTR channels. A reproducible and physiologically relevant human iPSC-derived platform for in vitro modeling of the neurocardiac junction
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