Toward Precision Medicine in the Treatment of Arrhythmogenic Cardiomyopathy

This review highlights the current lack of disease specific therapies available for arrhythmogenic cardiomyopathy (ACM) and explores recent new advances toward novel genotype specific therapies.

Although our understanding of ACM genetics has grown, current therapies are largely based on the general treatment strategies of arrhythmias and heart failure that do not target the diverse underlying pathogenic causes of ACM. Recently, there have been several efforts toward genotype-specific targeted therapies including a small molecule inhibitor for LMNA cardiomyopathy, three distinct gene replacement therapy approaches for PKP2 cardiomyopathy, as well as early work with base editing and prime editing on RBM20 cardiomyopathy.

There is currently a lack of ACM-specific therapies with minimal disease modifying treatments. Advances in gene replacement and editing offer hope for promising novel genotype-specific therapies with the goal of reversing the underlying disease process.