Antisense Oligonucleotide Therapy for Neurodevelopmental Disorders

We describe the molecular mechanisms of ASO treatment for four neurodevelopmental and neuromuscular disorders. The ASO nusinersen is a general treatment for mutations ofSMN1 in spinal muscular atrophy that corrects the splicing defect in theSMN2 gene. Milasen is a patient-specific ASO that rescues splicing ofCNL7 in Batten ’s disease. STK-001 is an ASO that increases expression of the sodium channel geneSCN1A by exclusion of a poison exon. An ASO that reduces the abundance of theSCN8A mRNA is therapeutic in mouse models of developmental and epileptic encephalopathy. These examples demonstrate the variety of mechanisms and range of applications of ASOs for treatment of neurodevelopmental disorders.Dev Neurosci
Source: Developmental Neuroscience - Category: Neuroscience Source Type: research