Author

T. Bozoglu

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Open access Sep 2026

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.

Petra Runa-Vochozkova, M. Vaškovičová, J. Cambra et al. · Lab animal · 0 citations

#gene editing Sep 2026

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.

Ina Luksch, Christine M. Poch, Aylin Mayer et al. · Human Gene Therapy · 0 citations