Antisense oligonucleotides: the next frontier for treatment of neurological disorders

Antisense oligonucleotides (ASOs) are short, synthetic, single-stranded oligodeoxynucleotides that can alter RNA and reduce, restore, or modify protein expression through several distinct mechanismsBy targeting the source of the pathogenesis, ASO-mediated therapies have an higher chance of success than therapies targeting downstream pathwaysAdvances in the understanding of ASO pharmacology have provided momentum for translating these therapeutics into the clinicTwo ASO-mediated therapies have received approval from the US Food and Drug Administration for the treatment of Duchenne muscular dystrophy and spinal muscular atrophyFurther advancement of ASOs in the clinic urgently requires optimization of ASO delivery, target engagement, and safety profileThis technology holds the potential to change the therapeutic landscape for many neurological and non-neurological conditions in the near future