RLR天然免疫信号通路在长期腺相关病毒感染中的作用及机制

The role and mechanism of RLR innate immune signaling pathway in long-term AAV infection.

作者信息

Wang Yixuan, Pei Xiaolei, Huang Weilin, Sima Helin, Yang Yuxiang, Xu Xiaopeng, Yang Xinyi, Ren Changhuai, Zhang Lei, Shao Wenwei

机构信息

Academy of Medical Engineering and Translational Medicine, Medical College, Tianjin University, Tianjin 300072, China; State Key Laboratory of Experimental Hematology of China, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, 288 Nanjing Road, Tianjin 300020, China.

State Key Laboratory of Experimental Hematology of China, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, 288 Nanjing Road, Tianjin 300020, China.

出版信息

Int Immunopharmacol. 2025 Apr 4;151:114267. doi: 10.1016/j.intimp.2025.114267. Epub 2025 Feb 20.

Abstract

Adeno-associated virus (AAV)-mediated gene therapy represents a promising approach for treating genetic disorders. However, challenges remain in achieving stable transgene expression and mitigating liver injury during long-term therapy. Previous studies have implicated the activation of RIG-I-like receptors (RLRs), which detect double-stranded RNA (dsRNA), as a potential inhibitor of transgene expression. In this study, we investigated the role of the RLR pathway in AAV-transduced cells, with a focus on the generation of sense and antisense RNA, as well as the formation of dsRNA. Our findings revealed that dsRNA is produced following AAV transduction, leading to the activation of the RLR pathway and the induction of innate immune responses. Prolonged AAV transduction in mice resulted in significant liver injury, which was independent of adaptive immune activation. Instead, mitochondrial antiviral signaling protein (MAVS) activation emerged as a critical mediator of these effects. Notably, downregulation of MAVS enhanced transgene expression, suggesting that modulating MAVS could enhance the efficacy of AAV-based gene therapy. This study elucidates the mechanisms underlying dsRNA formation and RLR pathway activation, highlighting their impact on the efficacy of AAV gene therapy. These findings suggest that strategies aimed at minimizing dsRNA production and targeting the RLR-MAVS pathway could reduce immune activation and enhance therapeutic transgene expression, thereby optimizing AAV-based interventions for genetic disorders.

摘要

腺相关病毒(AAV)介导的基因治疗是治疗遗传疾病的一种有前景的方法。然而,在长期治疗过程中,实现稳定的转基因表达和减轻肝损伤方面仍存在挑战。先前的研究表明,检测双链RNA(dsRNA)的视黄酸诱导基因I样受体(RLR)的激活是转基因表达的潜在抑制剂。在本研究中,我们研究了RLR途径在AAV转导细胞中的作用,重点关注正义和反义RNA的产生以及dsRNA的形成。我们的研究结果表明,AAV转导后会产生dsRNA,导致RLR途径的激活和先天免疫反应的诱导。在小鼠中长时间进行AAV转导会导致严重的肝损伤,这与适应性免疫激活无关。相反,线粒体抗病毒信号蛋白(MAVS)的激活成为这些效应的关键介质。值得注意的是,MAVS的下调增强了转基因表达,这表明调节MAVS可以提高基于AAV的基因治疗的疗效。本研究阐明了dsRNA形成和RLR途径激活的潜在机制,突出了它们对AAV基因治疗疗效的影响。这些发现表明,旨在最小化dsRNA产生并靶向RLR-MAVS途径的策略可以减少免疫激活并增强治疗性转基因表达,从而优化基于AAV的遗传疾病干预措施。

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