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

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. Th...

Descripción completa

Detalles Bibliográficos
Autores principales: Chand, Deepa H., Sun, Rui, Diab, Karim A., Kenny, Damien, Tukov, Francis Fonyuy
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10125853/
https://www.ncbi.nlm.nih.gov/pubmed/37095320
http://dx.doi.org/10.1038/s41434-023-00401-5

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10125853/
https://www.ncbi.nlm.nih.gov/pubmed/37095320
http://dx.doi.org/10.1038/s41434-023-00401-5

Ejemplares similares