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