ABSTRACT Background Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are X‐linked dystrophinopathies caused by mutations in the dystrophin (DMD) gene. A common DMD‐causing mutation in humans is exon 52 deletion (DMDΔ52), which disrupts the reading frame and abolishes dystrophin expression. Therapeu...
Sarah Reschke, Alexander Graf, F. Hadlich et al.· Journal of Cachexia, Sarcope...· 0 citations
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 shown that antisense oligonucleotide-mediated inhibition of miR-128-3p restores muscle mass and function in aged mice, improves cardiac function while limiting adverse remodeling following myocardial infarction, and ameliorates skeletal and cardiac muscle pathology in mouse and pig models of Duchenne muscular dys...
Melissa A. Boldridge, Lei Xu, Xiao-Yin Wang et al.· bioRxiv· 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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