Article

Perm1 Gene Therapy Mitigates PRDM16-Associated Cardiomyopathy

2026-04-15

Abstract excerpt

<h4>Background</h4> Pathogenic variants in PR domain containing 16 ( PRDM16 ) cause pediatric and adult cardiomyopathies characterized by ventricular dilation, systolic dysfunction, and impaired metabolic maturation. Cardiac deficiency of PRDM16 alters metabolic gene expression and long-chain fatty acid (FA) metabolites. However, the downstream mediators involved are not well characterized. Furthermore, whether...

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Literature Corpus work: 43de17a5-8fb6-5ee8-b582-97acb1cfc94c
DOI: 10.64898/2026.04.13.718330

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