Key result
Gene therapy approaches for hypertrophic cardiomyopathy show early promise in restoring sarcomeric biology, establishing HCM as an important model for cardiac gene therapy.
Why the study?
Hypertrophic cardiomyopathy has well-defined sarcomeric genetic causes that make it an attractive target for molecular gene therapy, but challenges remain in translating these approaches clinically.
Gene therapy represents a promising but complex emerging therapeutic avenue for modifying the underlying molecular and structural biology of hypertrophic cardiomyopathy.
Hypertrophic cardiomyopathy (HCM) is a prototypical inherited cardiomyopathy with well-defined sarcomeric genetic underpinnings that make it an attractive target for molecular therapy. We review recent advances in gene-based approaches for HCM, including adeno-associated virus-mediate gene replacement, allele-specific silencing, and emerging gene editing strategies, and highlight the first demonstrations of in vivo target engagement and early clinical translation. Early-phase studies suggest that restoration of sarcomeric biology can favorably impact molecular and structural disease features. We further discuss key challenges related to immune responses, delivery efficiency, response durability, and patient selection that will shape the next phase of development. Together, these developments establish HCM as one of the important early models for cardiac gene therapy and highlight both the promise and complexity of translating genetic insight into durable clinical benefit.
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Keen et al. (2026) conducted a review in Hypertrophic cardiomyopathy (HCM). Gene therapy approaches was evaluated. Gene therapy approaches for hypertrophic cardiomyopathy show early promise in restoring sarcomeric biology, establishing HCM as an important model for cardiac gene therapy.
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