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
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
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.
Maren Kirstin Schuhmacher, Christoph Gruber, Christopher M. R. Lang et al.· Nature· 1 citation
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