C. Kupatt

ALPK3 gene addition to restore sarcomeric quality control

A new bioorthogonal PET reporter gene for cardiac regenerative medicine binding to DTPA-lanthanide complexes

Activating a patient-relevant pathogenic variant of a genetically defined heart disease in a humanized pig model

The study describes the generation of a humanized pig model carrying an inducible phospholamban R14del mutation for cardiomyopathy modeling and demonstrates Cre-based activation strategies as efficient alternatives to germline Cre-driver lines in large animals.

Development of an AAV-Encoded Adenine Base Editor for Duchenne Muscular Dystrophy.

It is suggested that even modest levels of base editing-mediated exon skipping may ameliorate the DMD cardiac phenotype toward a BMD-like state, supporting the translational potential of this dual rAAV base editing approach for DMD cardiomyopathy.

Creating bottom-up RNA transfer vehicles from synthetic protein assemblies

This work demonstrates how proteins created by generative artificial intelligence can be harnessed for the rational engineering of RNA transport systems with the desired properties by overcoming the limitations of natural protein diversity.