ABSTRACT RNA interference (RNAi) is an ancient biological mechanism used to defend
against external invasion. It theoretically can silence any disease-related genes in a …

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 …

Transthyretin (TTR) is a tetrameric protein synthesized mostly by the liver and secreted into
the plasma. TTR molecules can misfold and form amyloid fibrils in the heart and peripheral …

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

Conjugation of small interfering RNA (siRNA) to an asialoglycoprotein receptor ligand
derived from N-acetylgalactosamine (GalNAc) facilitates targeted delivery of the siRNA to …

Significant progress has been made in the advancement of RNAi therapeutics by combining
a synthetic triantennary N-acetylgalactosamine ligand targeting the asialoglycoprotein …

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 …

Therapeutic gene silencing promises significant progress in pharmacotherapy, including
considerable expansion of the druggable target space and the possibility for treating orphan …

The highly specific induction of RNA interference-mediated gene knockdown, based on the
direct application of small interfering RNAs (siRNAs), opens novel avenues towards …

Small interfering RNA (siRNA) enables efficient target gene silencing by employing a RNA
interference (RNAi) mechanism, which can compromise gene expression and regulate gene …