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血栓性微血管病作为基于病毒载体的基因治疗新出现的并发症

Thrombotic Microangiopathy as an Emerging Complication of Viral Vector-Based Gene Therapy.

作者信息

Schwotzer Nora, El Sissy Carine, Desguerre Isabelle, Frémeaux-Bacchi Véronique, Servais Laurent, Fakhouri Fadi

机构信息

Service of Nephrology and Hypertension, Department of Medicine, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland.

Department of Immunology, Assistance Publique-Hôpitaux de Paris, Hôpital Européen Georges Pompidou, Paris, France.

出版信息

Kidney Int Rep. 2024 Apr 15;9(7):1995-2005. doi: 10.1016/j.ekir.2024.04.024. eCollection 2024 Jul.

DOI:10.1016/j.ekir.2024.04.024
PMID:39081755
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11284364/
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希望。尽管研究已显示基因治疗的疗效，但也出现了严重不良事件，包括基于病毒载体的基因治疗后发生的血栓性微血管病（TMA）。在本综述中，我们简要总结了基因治疗的概念以及病毒载体引发的免疫反应。我们还讨论了基因治疗相关TMA的发生率、表现及潜在的潜在机制，包括补体激活。需要进一步研究以更好地界定这种基因治疗严重并发症的发病机制以及预防它的最佳措施。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9a3/11284364/882e6424c12a/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9a3/11284364/a2921a79c045/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9a3/11284364/7bfa62d0271c/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9a3/11284364/882e6424c12a/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9a3/11284364/a2921a79c045/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9a3/11284364/7bfa62d0271c/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d9a3/11284364/882e6424c12a/gr3.jpg

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