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 …

Numerous chemically modified oligonucleotides have been developed so far and show their
own unique chemical properties and pharmacodynamic/pharmacokinetic characteristics …

Oligonucleotides (ONs) can interfere with biomolecules representing the entire extended
central dogma. Antisense gapmer, steric block, splice-switching ONs, and short interfering …

Conspectus Efforts to chemically modify nucleic acids got underway merely a decade after
the discovery of the DNA double helix and initially targeted nucleosides and nucleotides …

RNA plays a central role in the expression of all genes. Because any sequence within RNA
can be recognized by complementary base pairing, synthetic oligonucleotides and …

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

Oligonucleotide-based therapeutics have made rapid progress in the clinic for treatment of a
variety of disease indications. Unmodified oligonucleotides are polyanionic macromolecules …

Of the first generation oligonucleotide analogs, the class that has resulted in the broadest
range of activities and about which the most is known is the phosphorothioate class …

Antisense technology constitutes development of sequence-specific DNA or RNA analogs
that can block the activity of selected single-stranded genetic sequences and offer the …

Oligonucleotide therapeutics are a novel promising class of drugs designed to specifically
target either coding or non-coding RNA molecules to revolutionize treatment of various …