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