Key result
Gene therapy strategies offer potential breakthrough treatments for LMNA-related cardiomyopathy.
Why the study?
LMNA-related cardiac dysfunction is resistant to existing cardiac therapies, leaving a major unmet clinical need, while diverse disease mechanisms preclude small-molecule drugs from targeting all pathways.
Design
Review
Authors
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Does not support clinical use in LMNA cardiomyopathy; leaves open the need for trials of gene-based approaches.
Gene therapy represents a promising future therapeutic modality for LMNA-related cardiomyopathy, addressing the limitations of small-molecule drugs.
Wang et al. (2025) conducted a review in LMNA-related cardiomyopathy. Gene therapy was evaluated. Gene therapy strategies, including gene supplementation, silencing, and genome editing, offer potential breakthrough therapeutic modalities for LMNA-related cardiomyopathy.
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