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