PDF(Pubmed)
Abstract：
Gene therapy has brought tremendous hope for patients with severe life-threatening monogenic diseases. Although studies have shown the efficacy of gene therapy, serious adverse events have also emerged, including thrombotic microangiopathy (TMA) following viral vector-based gene therapy. In this review, we briefly summarize the concept of gene therapy, and the immune response triggered by viral vectors. We also discuss the incidence, presentation, and potential underlying mechanisms, including complement activation, of gene therapy-associated TMA. Further studies are needed to better define the pathogenesis of this severe complication of gene therapy, and the optimal measures to prevent it.
摘要：
基因治疗为患有严重危及生命的单基因疾病的患者带来了巨大的希望。虽然研究表明基因治疗的功效,也出现了严重的不良事件，包括基于病毒载体的基因治疗后的血栓性微血管病（TMA）。在这次审查中,我们简要总结了基因治疗的概念，和病毒载体引发的免疫反应。我们还讨论了发病率，介绍,和潜在的潜在机制，包括补体激活,基因治疗相关的TMA。需要进一步的研究来更好地定义这种基因治疗的严重并发症的发病机理，以及防止这种情况的最佳措施。
