Recent years have seen unprecedented activity in the development of RNA-silencing
oligonucleotide therapeutics for metabolic diseases. Improved oligonucleotide design and …

Systemic diseases of liver origin (SDLO) are complex diseases in multiple organ systems,
such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ …

Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …

RNA interference (RNAi) is a robust gene silencing mechanism that degrades mRNAs
complementary to the antisense strands of double-stranded, short interfering RNAs …

The therapeutic pathways that modulate transcription mechanisms currently include gene
knockdown and splicing modulation. However, additional mechanisms may come into play …

Antisense oligonucleotide (AON) therapies involve short strands of modified nucleotides that
target RNA in a sequence‐specific manner, inducing targeted protein knockdown or …

Synthetic antisense oligonucleotides (ASOs) and duplex RNAs (dsRNAs) are an
increasingly successful strategy for drug development. After a slow start, the pace of success …

In the last decade, the field of oligonucleotide therapeutics has matured, with the regulatory
approval of several single-stranded and double-stranded RNA drugs. In this Perspective, I …