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Article

Reducing Granules Without Splicing Restoration Alleviates RBM20 Cardiomyopathy

17 Apr 2025

Abstract excerpt

BACKGROUND: RBM20 (RNA binding motif protein 20) cardiomyopathy is a severe form of dilated cardiomyopathy (DCM). Genetic variants in the nuclear localization signal of Rbm20 hinder its nuclear import and promote cytoplasmic pathogenic RNP (ribonucleoprotein) granules. We aimed to investigate whether reducing RNP granules by inhibiting Rbm20 expression could alleviate the DCM phenotype in Rbm20 S639G ( Rbm20...

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PMID: 40242865
DOI: 10.1161/circresaha.124.324781

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