Antisense-Mediated Gene Therapy Targeting DMPK Restores Cardiac Ion Channel Function and Electrical Stability in Myotonic Dystrophy Type 1 - PubMed
5 hours ago
- #Cardiac Electrophysiology
- #Antisense Oligonucleotide
- #Myotonic Dystrophy Type 1
- Antisense oligonucleotide IONIS-486178 targets mutant DMPK transcripts in DM1 patient-derived hiPSC-CMs.
- Treatment achieves ~90% DMPK knockdown, reduces toxic nuclear foci, and corrects SCN5A mis-splicing.
- Functional rescue restores sodium, calcium, and potassium ionic currents and normalizes action potential kinetics.
- The therapy reduces arrhythmogenic events, supporting ASO targeting of DMPK for cardiac complication prevention.