Disease heterogeneity Dilated cardiomyopathy Intra-CSF delivery Errance diagnotique Extremely preterm infants GABA G-Secretase Calcium handling Microglia Long-term handicap Spinal muscular atrophy Dicer ERK1/2 signaling Adult patients DTI Clinical trial Maternal behavior Melatonin Longitudinal progression FTD SMN IUGR C9orf72 Modèle murin Amyotrophic Lateral Sclerosis Biomarker Chondrocytes Lentiviral vectors Inflammation AAV Duchenne Muscular Dystrophy Cell stemness Distal myopathy DPRs MiRNA Clinical trials Brain damage Brain MRI IPSCs Genetics Functional outcomes Biological marker FGR 3xTgAD Mice Les paramètres respiratoires Blood brain barrier Bone involvement Brain Gene transfer MND Aav10 MUNIX MRNP assembly Adult SMA AICD Maternal malnutrition CRISPR/SaCas9 Fetal growth restriction Disease modifiers FOXO3a Antisense oligonucleotides Clinical markers Glucocorticosteroid ASO Bioinformatics ASOs Genetical therapy Epigenetic changes Intra-uterine growth restriction Mecp2 Brain injury Albumin gene targeting Maladie neuromusculaire Skeletal muscle GeneRide Prematurity Coagulation factor IX Cellules souches musculaires Early-onset sepsis Brain development Neuromuscular disease Gene therapy Cartilage and bone regeneration Cell reprogramming Biomarkers Amyotrophie spinale CNS Effector T cells Fabry disease lysosomal storage disorders adeno asociated virus-9 ALS IRM MRI Bone development Adenosine Mouse model LMNA Icv Diseases Brain imaging Cofilin-1