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