Aug 2026
ALPK3 gene addition to restore sarcomeric quality control
Julian Grünewald, C. Kupatt
· Nature Cardiovascular Resear... · 0 citations
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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.
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