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