Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

Antisense technology is beginning to deliver on the broad promise of the technology. Ten
RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double …

The first published description of therapeutic applications of antisense oligonucleotide
(ASO) technology occurred in the late 1970s and was followed by the founding of …

Pharmacokinetic properties of oligonucleotides are largely driven by chemistry of the
backbone and thus are sequence independent within a chemical class. Tissue …

Dramatic advances in understanding of the roles RNA plays in normal health and disease
have greatly expanded over the past 10 years and have made it clear that scientists are only …

Recent studies have led to a greater appreciation of the diverse roles RNAs play in
maintaining normal cellular function and how they contribute to disease pathology …

Recent progress in understanding phosphorothioate antisense oligonucleotide (PS-ASO)
interactions with proteins has revealed that proteins play deterministic roles in the …

The genetic basis for most inherited neurodegenerative diseases has been identified, yet
there are limited disease-modifying therapies for these patients. A new class of drugs …

Advances in medicinal chemistry have produced new chemical classes of antisense
oligonucleotides (ASOs) with enhanced therapeutic properties. Conjugation of the …

Before a messenger RNA (mRNA) is translated into a protein in the cytoplasm, its pre-mRNA
precursor is extensively processed through capping, splicing and polyadenylation in the …