[en] 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.
Disciplines :
Pediatrics
Author, co-author :
Schwotzer, Nora; Service of Nephrology and Hypertension, Department of Medicine, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland
El Sissy, Carine; Department of Immunology, Assistance Publique-Hôpitaux de Paris, Hôpital Européen Georges Pompidou, Paris, France ; Paris University, Paris, France
Desguerre, Isabelle; Paediatric Neurology Department, Necker Hospital, APHP Centre, Université Paris Cité, Paris, France
Frémeaux-Bacchi, Véronique; Department of Immunology, Assistance Publique-Hôpitaux de Paris, Hôpital Européen Georges Pompidou, Paris, France ; Paris University, Paris, France
Servais, Laurent ; Université de Liège - ULiège > Département des sciences cliniques ; MDUK Oxford Neuromuscular Center and NIHR Oxford Biomedical Research Center, University of Oxford, Oxford, UK
Fakhouri, Fadi; Service of Nephrology and Hypertension, Department of Medicine, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland
Language :
English
Title :
Thrombotic Microangiopathy as an Emerging Complication of Viral Vector-Based Gene Therapy.
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