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

2022-09-01

Abstract excerpt

<title>Abstract</title> <p>Human gene 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 all c...

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Literature Corpus work
91886c04-3cf9-5244-99ca-6056965be911
DOI
10.21203/rs.3.rs-1979632/v1
Open publication

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Review of cardiac safety in onasemnogene abeparvovec gene therapy: translation from preclinical to clinical findingsDOI 10.21203/rs.3.rs-1979632/v1
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