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