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Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings

24 Apr 2023

Abstract excerpt

Human gene replacement therapies such as onasemnogene abeparvovec (OA) use recombinant adeno-associated virus (rAAV) vectors to treat monogenic disorders. The heart and liver are known target organs of toxicity in animals; with cardiac and hepatic monitoring recommended in humans after OA dosing. This manuscript provides a comprehensive description of cardiac data from preclinical studies and clinical sources...

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PMID: 37095320
DOI: 10.1038/s41434-023-00401-5

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