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 …

MicroRNAs (miRNAs) are evolutionarily conserved small non-coding RNAs that have crucial
roles in regulating gene expression. Increasing evidence supports a role for miRNAs in …

Oligonucleotides (ONs), and their chemically modified mimics, are now routinely used in the
laboratory as a means to control the expression of fundamentally interesting or …

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

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

Vascular endothelial growth factor (VEGF) has been implicated in the pathological induction
of new blood vessel growth in a variety of proliferative disorders. Using the SELEX process …

Two decades of research on RNA interference (RNAi) have transformed a breakthrough
discovery in biology into a robust platform for a new class of medicines that modulate mRNA …

In an effort to discover novel oligonucleotide modifications for antisense therapeutics, we
have prepared oligodeoxyribonucleotides containing more than 200 different modifications …

The majority of the human genome encodes RNAs that do not code for proteins. These non-
coding RNAs (ncRNAs) affect normal expression of the genes, including oncogenes and …

RNA interference (RNAi) is a remarkable endogenous regulatory pathway that can bring
about sequence-specific gene silencing. If harnessed effectively, RNAi could result in a …