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Pathogenic variants in FLNC encoding filamin C have been firstly reported to cause myopathies, and were recently linked to isolated cardiac phenotypes. Our aim was to estimate the prevalence of FLNC pathogenic variants in subtypes of cardiomyopathies and to study the relations between phenotype and genotype. DNAs from a cohort of 1150 unrelated index-patients with isolated cardiomyopathy (700 hypertrophic, 300 dilated, 50 restrictive cardiomyopathies, and 100 left ventricle non-compactions) have been sequenced on a custom panel of 51 cardiomyopathy disease-causing genes. An FLNC pathogenic variant was identified in 28 patients corresponding to a prevalence ranging from 1% to 8% depending on the cardiomyopathy subtype. Truncating variants were always identified in patients with dilated cardiomyopathy, while missense or in-frame indel variants were found in other phenotypes. A personal or family history of sudden cardiac death (SCD) was significantly higher in patients with truncating variants than in patients carrying missense variants (P = .01). This work reported the first observation of a left ventricular non-compaction associated with a unique probably causal variant in FLNC which highlights the role of FLNC in cardiomyopathies. A correlation between the nature of the variant and the cardiomyopathy subtype was observed as well as with SCD risk.
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http://dx.doi.org/10.1111/cge.13594 | DOI Listing |
Eur Heart J
August 2025
Myocardial Homeostasis and Cardiac Injury Programme, Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain.
Background: Truncating variants in the Filamin C gene (FLNCtv) are a frequent cause of genetic dilated cardiomyopathy (DCM) and non-dilated left ventricular cardiomyopathy (NDLVC), both characterized by arrhythmic complications and increased risk of sudden cardiac death. Currently, no gene-specific therapies exist for FLNCtv-induced cardiomyopathy. CRISPR activation (CRISPRa), which upregulates gene expression via transcriptional activation without cutting the genome, offers a promising strategy, particularly for genes like FLNC whose large size precludes conventional AAV-based gene replacement.
View Article and Find Full Text PDFTrends Cardiovasc Med
July 2025
Department of Medicine, University of Udine, Italy; Cardiothoracic Department, University Hospital Santa Maria della Misericordia, ASUFC, Udine, Italy. Electronic address:
Inflammatory cardiomyopathy (CMP) is a myocardial disorder characterized by persistent inflammation leading to ventricular dysfunction and remodeling, often evolving from acute myocarditis of infectious or immune-mediated origin. Its pathogenesis is multifactorial, involving viral triggers, dysregulated immune responses, and genetic predispositions, resulting in a broad clinical spectrum-from infarct-like presentations and arrhythmias to heart failure presentations. Diagnosis requires an integrated approach using multiple diagnostic tools, integrating clinical evaluation, ECG, biomarkers, multimodality imaging, including cardiac magnetic resonance (CMR), and endomyocardial biopsy (EMB), which remains the gold standard for identifying histologic subtypes and guiding immunosuppressive therapy.
View Article and Find Full Text PDFCase Rep Med
June 2025
Cardiology Service, Fundación Valle del Lili, Cali, Colombia.
Dilated cardiomyopathy is a leading cause of heart failure and heart transplantation. Among its etiologies, genetic variants account for up to 35% of cases. Variants in the gene have gained recognition due to their association with a higher risk of major ventricular arrhythmias and sudden cardiac death.
View Article and Find Full Text PDFCell Rep
June 2025
Laboratory of Pathogen Microbiology and Immunology, Institute of Microbiology, Chinese Academy of Sciences, Beijing 100101, China; Medical School, University of Chinese Academy of Sciences, Beijing 101408, China. Electronic address:
Dissecting host factors critical for viral infection and understanding their mechanisms of action is critical for identifying drug targets. Here, we leverage a genome-wide CRISPR base-editing screen to identify functional lysine residues in host factors required for influenza A virus (IAV) replication. Multiple host genes, including GSTM4, FLNC, HMGB1, ZNF236, GRIP1, and PXN, along with regulatory lysine codons, are identified.
View Article and Find Full Text PDFActa Neuropathol Commun
May 2025
Department of Neuroimmunology and Neuromuscular Diseases, Fondazione IRCCS Neurological Institute Carlo Besta, Muscle Cell Biology Lab, Via Amadeo 42, 20133, Milano, Italy.
Myofibrillar Myopathies (MFMs) are a growing group of muscular disorders genetically determined, whose diagnosis is based on histological features as myofibrillar degeneration, Z-disk disorganization and protein aggregates' accumulation. Protein aggregates that do not fit the proteasome's narrow pore are targeted for removal via a specialized form of autophagy, called aggrephagy. Our study aims to investigate the potential pathogenic role of aggrephagy in 52 muscle samples from an Italian MFM multicentric cohort.
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