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Mutated Genes Causing Inherited Cardiomyopathies
- Molecular classification
- Gene, Sarcomeric protein, RNA-binding protein
01
Overview
Mutated genes responsible for inherited cardiomyopathies represent therapeutic targets for gene replacement or augmentation strategies. These therapies aim to correct the underlying genetic defect by introducing a functional copy of the gene or enhancing expression of the existing healthy allele. Common targets include MYBPC3 in hypertrophic cardiomyopathy and RBM20 in dilated cardiomyopathy.
- Other names
- Cardiomyopathy GenesHCM GenesDCM Genes
02
Mechanism of action
Gene replacement or augmentation therapy; introduction of functional gene copy or enhancement of existing expression
03
Biological functions
Cardiac muscle contractionRNA splicingGene expression regulation
04
Disease associations
Hypertrophic CardiomyopathyDilated CardiomyopathyInherited CardiomyopathyHeart Failure
05
Safety considerations
Immune response to viral vectorsOff-target effects with genome editingDelivery efficiency to cardiac tissueLong-term safety of gene editing
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