AAV VECTOR Therapy Treatment RNA interference Cardiovascular disease Heart Heart failure Centronuclear myopathy Joint laxity IPSC Cardiac conduction system Angiotensin-converting enzyme inhibitors Gene therapy Allele‐specific silencing therapy CMTX Neuromuscular diseases Actionability Dilated cardiomyopathy Muscle BiP Exome Next generation sequencing Emerin Treatment delay Muscular dystrophy MD COL6A1 Regeneration Myogenesis Becker muscular dystrophy Butyrylcholinesterase LMNA Becker muscular dystrophy BMD Duchenne muscular dystrophy DMD miRNA nNOS Lamins GNE Duchenne muscular dystrophy Errance diagnostique Laminopathie Angiotensin-converting enzyme inhibitor Dystrophine POPDC1 Biomarker Laminopathy Dystrophie musculaire C elegans Lamin A/C nuclei Connective tissue Hypermobile EDS Cardiomyopathy Emery-Dreifuss muscular dystrophy Patient registry A-type lamin Clinical trial Allele-specific silencing LGMD Acetyltransferase Ehlers‐Danlos Syndrome Autophagosome maturation Lamin A/C Base de données FAIR CRISPR Allele-specific silencing therapy Cancer biomarkers Lamin A/C LMNA gene CSF protein COVID-19 Muscular dystrophy Diagnosis Myotubes Calcium handling Myopathies Nuclear envelope A-type lamins Cell biology Cardiology Biological sciences Laminopathies AAV COL1A1 Maladies rares et orphelines BVES Mutations COL6A3 Collagen VI-related myopathies NGS collagen type VI congenital muscular dystrophy CMD limb-girdle muscular dystrophy LGMD muscular MRI neuromuscular disorders Adult SMA Cancer LMNA gene C2C12 LMNA-related congenital muscular dystrophy Titin Skeletal muscle Mouse Myologie Actionable gene Maladies rares Dynamin 2 INPP5K Alternative splicing Myopathy Congenital muscular dystrophy Rare neuromuscular diseases Rare diseases