RNA-Based Therapies for Treating Monogenic Cardiomyopathies.

As the field of cardiovascular medicine advances toward precision and personalized care, ribonucleic acid (RNA)-based therapies have emerged as powerful tools with the potential application for monogenic cardiomyopathies. Multiple classes of RNA-based therapeutics are currently being investigated, including antisense oligonucleotides, microRNAs, small interfering RNAs, small hairpin RNAs, and long noncoding RNAs. Recent progress in viral and nonviral delivery systems has enhanced the feasibility of these approaches. These advances are particularly relevant to a growing list of genetic disorders such as Duchenne muscular dystrophy, hypertrophic cardiomyopathy, titinopathies, and diseases linked to nuclear cytoskeletal or ion channel genes. Ongoing translational research will be critical to overcoming current challenges in durability, immune response, and delivery, paving the way for RNA therapeutics to become a cornerstone of precision cardiovascular medicine.