For the roughly one in 500 adults carrying a pathogenic variant linked to inherited cardiomyopathy, current management remains largely symptomatic — beta-blockers, defibrillators, transplant waitlists. A detailed review published in the Canadian Journal of Cardiology maps the emerging therapeutic landscape that could reframe these conditions as correctable molecular defects rather than lifelong sentences.

The review organizes gene-based strategies into three mechanistic pillars: gene replacement, gene silencing, and direct genome editing. Within editing, it traces a clear technological lineage — from foundational CRISPR-Cas9, which enables cuts at targeted genomic loci, to base editors capable of single-nucleotide conversion without double-strand breaks, to prime editors that can execute insertions, deletions, and both transition and transversion mutations with greater precision. On the delivery side, novel adeno-associated virus capsids are highlighted for reduced immunogenicity and improved myocardial transduction efficiency, alongside virus-like particles and lipid nanoparticles. Antisense oligonucleotides round out the toolkit by enabling exon-skipping strategies suited to large, mutation-dense genes such as TTN, which encodes the giant sarcomeric protein titin.

The significance of this convergence deserves careful framing. Cardiomyopathy genetics are heterogeneous — hypertrophic, dilated, arrhythmogenic, and restrictive subtypes each harbor distinct mutational architectures — meaning no single editing platform will suffice universally. CRISPR-based trials have reached clinical phases in other monogenic diseases, but cardiac delivery poses unique obstacles: the heart is post-mitotic, making homology-directed repair inefficient, and immune responses to viral capsids remain a serious safety variable. Prime editing's theoretical precision advantage is real, yet off-target cardiac data in humans remain sparse. This review reads as confirmatory and synthetic rather than paradigm-shifting — its value lies in consolidating a fast-moving field for practicing cardiologists. Adults with a family history of cardiomyopathy should view this trajectory as meaningful but early-stage; genetic testing today enables identification of at-risk individuals, positioning them to benefit from therapies that may reach broad clinical use within a decade.