Article

Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator

2023-05-18

Abstract excerpt

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-re...

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Literature Corpus work: b6e257a1-140e-5677-9fec-4267c33f4e5d
DOI: 10.1101/2023.05.16.23289881

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