Antisense Oligonucleotides Mediated Therapy for Neurodegenerative Disease

Abstract

Many neurodegenerative diseases like Alzheimer’s disease, Huntingtion’s disease, Duchenne’s muscular dystrophy and spinal muscular atrophy are linked to aggregated, toxic proteins. Antisense oligonucleotide-based strategies (ASOs) are the most direct method of targeting gene expression. Synthetic oligonucleotides bind to the target mRNA by Watson-Crick hybridization and can either promote the degradation of RNA or inhibit it. In 2016, two ASO therapies for spinal muscular atrophy and Duchenne muscular dystrophy were approved by the FDA.