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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 rec
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ef6ba766ff77731f7031f55d86feec8b
https://doi.org/10.21203/rs.3.rs-1979632/v1
https://doi.org/10.21203/rs.3.rs-1979632/v1