RNA-targeted therapies represent a platform for drug discovery involving chemically
modified oligonucleotides, a wide range of cellular RNAs, and a novel target-binding motif …

A main challenge in realizing the full potential of nucleic acid therapeutics is efficient
delivery of them into targeted tissues and cells. N-acetylgalactosamine (GalNAc) is a well …

ABSTRACT Introduction Antisense oligonucleotides (ASOs) have emerged as a promising
novel drug modality that aims to address unmet medical needs. A record of six ASO drugs …

Transthyretin (TTR) is a tetrameric protein synthesized primarily by the liver. TTR can misfold
into pathogenic ATTR amyloid fibrils that deposit in the nerves and heart, causing a …

Aims Amyloidogenic transthyretin (ATTR) amyloidosis is a fatal disease characterized by
progressive cardiomyopathy and/or polyneuropathy. AKCEA‐TTR‐LRx (ION‐682884) is a …

Oligonucleotide therapeutics hold promise for the treatment of muscle-and heart-related
diseases. However, oligonucleotide delivery across the continuous endothelium of muscle …

The potential of antisense oligonucleotides in gene silencing was discovered over 40 years
ago, which resulted in the growing interest in their chemistry, mechanism of action, and …

Oligonucleotides (OGNs) are relatively new modalities that offer unique opportunities to
expand the therapeutic targets. Reliable and high-throughput bioanalytical methods are …

ANGPTL3 has emerged as a therapeutic target whose inhibition results in profound
reductions of plasma lipids, including atherogenic triglyceride-rich lipoproteins and low …

A compact and stable bicyclic bridged ketal was developed as a ligand for the
asialoglycoprotein receptor (ASGPR). This compound showed excellent ligand efficiency …