Key result
Mutations in the MYBPC3 gene primarily cause haploinsufficiency and an increase in calcium sensitivity, leading to hypertrophic and dilated cardiomyopathies.
This review highlights the molecular mechanisms by which MYBPC3 mutations contribute to cardiomyopathy and heart failure, emphasizing haploinsufficiency and altered calcium sensitivity.
Warrants cautious MYBPC3 variant interpretation in clinic; leaves open targeted therapy trials.
Myosin binding protein C (MYBPC) is a crucial component of the sarcomere and an important regulator of muscle function. While mutations in different myosin binding protein C (MYBPC) genes are well known causes of various human diseases, such as hypertrophic (HCM) and dilated (DCM) forms of cardiomyopathy as well as skeletal muscular disorders, the underlying molecular mechanisms remain not well understood. A variety of MYBPC3 (cardiac isoform) mutations have been studied in great detail and several corresponding genetically altered mouse models have been generated. Most MYBPC3 mutations may cause haploinsufficiency and with it they may cause a primary increase in calcium sensitivity which is potentially able to explain major features observed in HCM patients such as the hypercontractile phenotype and the well known secondary effects such as myofibrillar disarray, fibrosis, myocardial hypertrophy and remodelling including arrhythmogenesis. However the presence of poison peptides in some cases cannot be fully excluded and most probably other mechanisms are also at play. Here we shall discuss MYBPC interacting proteins and possible pathways linked to cardiomyopathy and heart failure.
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Ralph Knöll (2011) conducted a review in Hypertrophic cardiomyopathy and dilated cardiomyopathy. Mutations in the MYBPC3 gene primarily cause haploinsufficiency and an increase in calcium sensitivity, leading to hypertrophic and dilated cardiomyopathies.
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