基因治疗中的病毒与非病毒载体：现状与临床前景

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基因治疗中的病毒与非病毒载体：现状与临床前景

英文原题：Viral and non-viral vectors in gene therapy: current state and clinical perspectives.

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Viral and non-viral vectors in gene therapy: current state and clinical perspectives.

PubMed 2025/07/01（内容时间） EBioMedicine Q1 · IF 11.2（JCR 2025）

技术：CAR-T 技术：基因治疗地区：中国

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研究概要

基因治疗的进步在治疗人类疾病方面取得了重要里程碑，为选择有限的患者带来了新的希望。

中文摘要

基因治疗技术的进步在治疗人类疾病方面取得了重要里程碑，为选择有限的患者带来了新的希望。这一进展的关键在于载体，包括病毒性和非病毒性方法，它们影响着基因治疗的成功。过去二十年中，三种广泛使用的病毒载体——慢病毒（LV）、腺病毒（Ad）和腺相关病毒（AAV）——促成了显著的临床前和临床成功，包括获批用于遗传性视网膜疾病的Luxturna和用于血液癌症的CAR-T疗法。最近，首个用于遗传性听力损失的人体双AAV疗法克服了大基因递送的难题，展示了患者听觉功能的恢复。此外，脂质纳米颗粒（LNP）和N-乙酰半乳糖胺（GalNAc）等非病毒载体也促成了成功的基因治疗产品。本综述聚焦于基因治疗中的病毒性和非病毒性递送系统，强调其在治疗人类疾病方面的现状和未来前景。

展开英文摘要原文

Advancements in gene therapy have achieved significant milestones in treating human diseases, offering renewed hope to patients with limited options. Key to this progress are vectors, which include both viral and non-viral methodologies that impact the success of gene therapy. Over the past two decades, three widely used viral vectors-lentiviruses (LV), adenoviruses (Ad), and adeno-associated viruses (AAV)-have enabled notable preclinical and clinical successes, including the approval of Luxturna for a genetic retinal disease and CAR-T therapies for blood cancers. Recently, the first-in-human dual AAV therapy for hereditary hearing loss, which overcomes large gene delivery, has showcased the restoration of auditory function for patients. Additionally, non-viral vectors such as lipid nanoparticles (LNP) and N-acetylgalactosamine (GalNAc) have led to successful gene therapy products. This review focuses on both viral and non-viral delivery systems in gene therapy, highlighting their current state and future perspectives in treating human diseases.

论文信息

作者: Geng G、Xu Y、Hu Z、Wang H、Chen X、Yuan W、Shu Y
第一作者单位: ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Fudan University, Shanghai, China; Institutes of Biomedical Sciences, Fudan University, Shanghai, China; State Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; MOE Engineering Research Center of Gene Technology, China.China
通讯作者单位: ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Fudan University, Shanghai, China; Institutes of Biomedical Sciences, Fudan University, Shanghai, China; State Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; MOE Engineering Research Center of Gene Technology, China. Electronic address: yilai_shu@fudan.edu.cn.China
文献类型: 综述
期刊: EBioMedicine2025 Aug

原文标识: PubMed 40602323　·　DOI 10.1016/j.ebiom.2025.105834

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