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