Key result
Direct interaction between cMyBP-C and Fhod3 directs Fhod3 localization to the C-zone, and aberrant Fhod3 mislocalization exacerbates the cardiomyopathic phenotype in cMyBP-C-null mice.
Fhod3 directly interacts with cMyBP-C and its mislocalization contributes to the pathogenesis of cMyBP-C-related hypertrophic cardiomyopathy.
Hypothesis-generating for Fhod3 targeting in cMyBP-C HCM; leaves open clinical translation.
Mutations in cardiac myosin-binding protein C (cMyBP-C) are a major cause of familial hypertrophic cardiomyopathy. Although cMyBP-C has been considered to regulate the cardiac function via cross-bridge arrangement at the C-zone of the myosin-containing A-band, the mechanism by which cMyBP-C functions remains unclear. We identified formin Fhod3, an actin organizer essential for the formation and maintenance of cardiac sarcomeres, as a cMyBP-C-binding protein. The cardiac-specific N-terminal Ig-like domain of cMyBP-C directly interacts with the cardiac-specific N-terminal region of Fhod3. The interaction seems to direct the localization of Fhod3 to the C-zone, since a noncardiac Fhod3 variant lacking the cMyBP-C-binding region failed to localize to the C-zone. Conversely, the cardiac variant of Fhod3 failed to localize to the C-zone in the cMyBP-C-null mice, which display a phenotype of hypertrophic cardiomyopathy. The cardiomyopathic phenotype of cMyBP-C-null mice was further exacerbated by Fhod3 overexpression with a defect of sarcomere integrity, whereas that was partially ameliorated by a reduction in the Fhod3 protein levels, suggesting that Fhod3 has a deleterious effect on cardiac function under cMyBP-C-null conditions where Fhod3 is aberrantly mislocalized. Together, these findings suggest the possibility that Fhod3 contributes to the pathogenesis of cMyBP-C-related cardiomyopathy and that Fhod3 is critically involved in cMyBP-C-mediated regulation of cardiac function via direct interaction.
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Matsuyama et al. (2018) studied Familial hypertrophic cardiomyopathy. Fhod3 overexpression or reduction was evaluated on Interaction and localization of Fhod3 and cMyBP-C, and effect on cardiomyopathic phenotype. Direct interaction between cMyBP-C and Fhod3 directs Fhod3 localization to the C-zone, and aberrant Fhod3 mislocalization exacerbates the cardiomyopathic phenotype in cMyBP-C-null mice.
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