The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …

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 …

Polymeric micelles are demonstrating high potential as nanomedicines capable of
controlling the distribution and function of loaded bioactive agents in the body, effectively …

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 …

The targeted and efficiency-oriented delivery of (therapeutic) nucleic acids raises hope for
successful gene therapy, ie, for the local and individual treatment of acquired and inherited …

Since the elucidation of the molecular structure of nucleic acids and understanding of their
role in encoding and translating genetic information into biological function, 1 the knowledge …

Nanotechnology has been widely used in the development of new strategies for drug
delivery and cancer therapy. Compared to traditional drug delivery systems, nano-based …

Triantennary N-acetyl galactosamine (GalNAc, GN3), a high-affinity ligand for the hepatocyte-
specific asialoglycoprotein receptor (ASGPR), enhances the potency of second-generation …

Gene therapy is the process of introducing foreign genomic materials into host cells to elicit
a therapeutic benefit. Although initially the main focus of gene therapy was on special …

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 …