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This Viewpoint explores the potential for genetic therapies to treat inherited cardiomyopathies at the intersection of unmet clinical need, patient appetite, and technological advances.
The mechanisms and current uses of gene upregulation strategies are reviewed and their advantages and limitations are critically evaluated as therapies for inherited cardiomyopathies.
Findings indicate that uORF-mediated repression of TTN is context-dependent and not recapitulated in a disease-relevant model, which highlights the complex regulation of TTN expression and the importance of validating regulatory elements in their endogenous context.
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