Opportunities and challenges for antisense oligonucleotide therapies

Kuijper EC, Bergsma AJ, Pim Pijnappel WWM, Aartsma-Rus A. Opportunities and challenges for antisense oligonucleotide therapies. J Inherit Metab Dis 2020   Abstract Antisense oligonucleotide (AON) therapies involve small strands of modified nucleotides that target RNA in a sequence-specific manner, inducing targeted protein knockdown or restoration. Currently, 10 AON therapies have been approved in the […]