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