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RNA-Based Therapies for Treating Monogenic Cardiomyopathies
Jonathan J Edwards1, Jonathan B Edelson2, Joseph Rossano1
1Department of Pediatrics, Division of Cardiology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA; Cardiovascular Institute, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
The Canadian Journal of Cardiology
|January 9, 2026
Summary
Ribonucleic acid (RNA)-based therapies show promise for genetic heart conditions like cardiomyopathies. Advances in delivery systems are key to overcoming challenges and enabling precision cardiovascular medicine.
Area of Science:
- Cardiovascular Medicine
- Genetics
- Molecular Biology
Background:
- Precision cardiovascular medicine is advancing rapidly.
- Monogenic cardiomyopathies are a target for new genetic therapies.
- Ribonucleic acid (RNA)-based therapeutics offer novel treatment strategies.
Purpose of the Study:
- To review the potential of various RNA-based therapeutics for monogenic cardiomyopathies.
- To highlight recent progress in RNA delivery systems.
- To identify challenges and future directions for RNA therapeutics in cardiovascular medicine.
Main Methods:
- Review of current literature on RNA-based therapies.
- Analysis of different classes of RNA therapeutics (e.g., antisense oligonucleotides, microRNAs, siRNAs, shRNAs, lncRNAs).
- Evaluation of viral and non-viral delivery systems.
Main Results:
- Multiple RNA therapeutic classes are under investigation for genetic cardiomyopathies.
- Progress in delivery systems enhances the feasibility of RNA-based treatments.
- Applications extend to genetic disorders like Duchenne muscular dystrophy and hypertrophic cardiomyopathy.
Conclusions:
- RNA therapeutics hold significant potential for treating monogenic cardiomyopathies.
- Overcoming challenges in durability, immune response, and delivery is crucial.
- RNA-based therapies are poised to become central to precision cardiovascular medicine.
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