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