Dysregulated iron homeostasis in dystrophin-deficient cardiomyocytes: correction by gene editing and pharmacological treatment.
Kalina AndrysiakGabriela MachajDominik PriesmannOlga WoźnickaAlicja MartyniakGuillem YllaMarcus KrügerElżbieta PyzaAnna Potulska-ChromikAnna Kostera-PruszczykAgnieszka ŁobodaJacek StępniewskiJozef DulakPublished in: Cardiovascular research (2023)
DMD-related cardiomyopathy is associated with the occurrence of iron overload and the resulting oxidative stress and mitochondrial damage. Abnormalities in iron homeostasis maintenance and the resulting iron overload may explain previously observed pathological features of DMD-associated cardiomyopathy, such as increased oxidative stress, and serve as a starting point for the search for new therapeutic strategies. Deferoxamine and pioglitazone, FDA-approved drugs for the treatment of iron overload and diabetes, respectively, demonstrated a positive effect on the reduction of ROS level in dystrophin-deficient cardiomyocytes; therefore, we believe that it would be beneficial to continue the studies to validate their effectiveness and safety in the context of DMD.