Kleopatra Rapti

The paper describes an ex vivo study using normothermic machine-perfused human hearts to evaluate AAV-mediated gene delivery for cardiac gene therapy.

This study uses base editors and AAVMYO to correct Rbm20 mutations in cardiomyocytes, improving splicing and cardiac function in mouse models of dilated cardiomyopathy.

This paper develops a cell-specific CRISPR/Cas9 activation system using miRNA-regulated anti-CRISPR proteins, demonstrating targeted gene activation in liver and muscle cells.