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- W4377014016 abstract "An N-of-1 trial was developed to deliver a dCas9-VP64 transgene designed to upregulate the cortical dystrophin as a custom therapy for a Duchenne muscular dystrophy (DMD) patient. After showing signs of mild cardiac dysfunction and pericardial effusion, the patient acutely decompensated and sustained cardiac arrest six-days after dosing and succumbed two-days later. Post-mortem examination revealed severe acute-respiratory distress syndrome with diffuse alveolar damage. Vector biodistribution data was obtained and revealed minimal expression of transgene in liver. There was no evidence of AAV9 antibodies nor of effector T cell reactivity. These findings demonstrate innate immune signaling with capillary leak as a form of toxicity in an advanced DMD case treated with high-dose rAAV gene therapy." @default.
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- W4377014016 date "2023-05-18" @default.
- W4377014016 modified "2023-10-03" @default.
- W4377014016 title "Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator" @default.
- W4377014016 doi "https://doi.org/10.1101/2023.05.16.23289881" @default.
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