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