Antisense Oligonucleotide Therapy for Neurodevelopmental Disorders - Hill, Sophie F; Meisler, Miriam H (Developmental neuroscience, 2021)
Keywords: Animals; Disease Models, Animal; Humans; Mice; Muscular Atrophy, Spinal - genetics; Muscular Atrophy, Spinal - therapy; NAV1.1 Voltage-Gated Sodium Channel; Neurodevelopmental Disorders - genetics; Neurodevelopmental Disorders - therapy; Oligonucleotides, Antisense; RNA Splicing - genetics; Antisense oligonucleotide; Gene therapy; SCN8A; Dravet syndrome; Spinal muscular atrophy