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Gene therapy for hypertrophic cardiomyopathy: From feasibility to a fieldwide framework
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DOI：10.1016/j.medj.2026.101229.png)
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Abstract
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The emergence of gene-based therapies for hypertrophic cardiomyopathy (HCM) represents a pivotal inflection point in cardiovascular medicine, but their successful translation will depend on coordinated advances beyond vector design alone. Here, we propose a fieldwide framework for HCM gene therapy development, encompassing core evaluation, safety and immunosuppression management, endpoint definition, long-term surveillance, and considerations of equity and scalability. We argue that harmonization across these domains is essential to ensure that early clinical signals are interpretable, comparable, and translatable into durable patient benefit. This framework provides a roadmap to guide the transition from early feasibility to broad, equitable implementation.
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