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 …

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

Therapeutics based on short interfering RNAs (siRNAs) delivered to hepatocytes have been
approved, but new delivery solutions are needed to target additional organs. Here we show …

The RNA interference (RNAi) pathway regulates mRNA stability and translation in nearly all
human cells. Small double-stranded RNA molecules can efficiently trigger RNAi silencing of …

Carbohydrates are essential mediators of many processes in health and disease. They
regulate self-/non-self-discrimination, are key elements of cellular communication, cancer …

Short-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide
variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease …

Targeted delivery of oligonucleotides to liver hepatocytes using N-acetylgalactosamine
(GalNAc) conjugates that bind to the asialoglycoprotein receptor has become a …

RNA-based therapies have catalyzed a revolutionary transformation in the biomedical
landscape, offering unprecedented potential in disease prevention and treatment. However …

Small interfering RNAs (siRNAs) acting via RNA interference mechanisms are able to
recognize a homologous mRNA sequence in the cell and induce its degradation. The main …

Small interfering RNAs (siRNAs) conjugated to a trivalent N-acetylgalactosamine (GalNAc)
ligand are being evaluated in investigational clinical studies for a variety of indications. The …