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