Article

Study design and rationale of Boxed-Breathing-Heart: a translational ex vivo study evaluating virus-mediated gene delivery for gene therapy in normothermic machine-perfused human hearts

2026-07-14

Abstract excerpt

Research on targeted genetic therapies for myocardial diseases, such as cardiomyopathies, currently focuses on (r)AAVs as the delivery method. Despite substantial efforts and advances in animal trials, predicting biodistribution and transduction efficacy in human tissue remains challenging due to interspecies differences in tissue tropism and the difficulty of accurately assessing alternative delivery routes and v...

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Literature Corpus work: 907f8387-7299-51e9-b619-778d0ea6fab5
DOI: 10.64898/2026.07.13.26357866

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