Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy
| العنوان: | Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy |
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| المؤلفون: | Julia Guillou, Alice de Pellegars, Florence Porcheret, Véronique Frémeaux-Bacchi, Emma Allain-Launay, Camille Debord, Manon Denis, Yann Péréon, Christine Barnérias, Isabelle Desguerre, Gwenaëlle Roussey, Sandra Mercier |
| المصدر: | Blood Advances. 6:4266-4270 |
| بيانات النشر: | American Society of Hematology, 2022. |
| سنة النشر: | 2022 |
| مصطلحات موضوعية: | Muscular Atrophy, Spinal, Fatal Outcome, Thrombotic Microangiopathies, Humans, Infant, Genetic Therapy, Immunotherapy, Hematology, Dependovirus |
| الوصف: | Adeno-associated virus (AAV) gene therapies are highly promising, such as the onasemnogene abeparvovec (Zolgensma) in spinal muscle atrophy (SMA). We report the first case of fatal systemic thrombotic microangiopathy (TMA) following onasemnogene abeparvovec in a 6-month-old child with SMA type 1, carrying a potential genetic predisposition in the complement factor I gene. Other cases of TMA have recently been reported after onasemnogene abeparvovec and after AAV9 minidystrophin therapy in Duchenne muscular dystrophy. The risk-benefit ratio of this therapy must therefore be assessed. Early recognition of TMA and targeted immunotherapy are fundamental to ensure the safety of patients treated with AAV gene therapies. |
| تدمد: | 2473-9537 2473-9529 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e09659c665eb0a757bbda768ebeb8911 https://doi.org/10.1182/bloodadvances.2021006419 |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....e09659c665eb0a757bbda768ebeb8911 |
| قاعدة البيانات: | OpenAIRE |
Full Text via ClinicalKey | تدمد: | 24739537 24739529 |
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