May 15, 2023 · Here we show the use of structure-guided rational design in a series of mouse and non-human primate studies to optimize a GalNAc-Lipid nanoparticle that allows for low-density …

Unlike complicated LNP formulations, GalNAc-siRNA conjugates are a simpler, smaller, and compositionally defined approach for hepatic delivery. A complete GalNAc-siRNA can be …

N-Acetylglucosamine (GlcNAc) is an amide derivative of the monosaccharide glucose. It is a secondary amide between glucosamine and acetic acid. It is significant in several biological …

Lipid nanoparticles (LNPs) are chemically synthesized multicomponent lipid formulations (~100 nm in size) encapsulating siRNAs for delivery to the target tissue. En route to their destination, …

An alternative approach to hepatic LNP uptake mediated by endogenous ApoE/LDLR binding is to use a multi-valent N-acetylgalactosamine (GalNAc) targeting ligand, which allows

Jan 15, 2017 · The hydrodynamic diameters of most monosaccharide-coated LNPs are in the range between 190 and 210 nm with relatively sharp distributions, while GlcNAc-LNP has a …

To better understand LDN biosynthesis and regulation, we stably expressed the cDNA encoding the Caenorhabditis elegans β1,4- N -acetylgalactosaminyltransferase (GalNAcT), which …

Nov 7, 2024 · In this review, we describe the characterization and biochemistry of OGT and OGA, cellular O-GlcNAc regulation, development of OGT and OGA inhibitors, O-GlcNAc in …

Unlike the complex glycans decorating the cell surface, the O-linked β- N -acetyl glucosamine (O-GlcNAc) modification is a simple intracellular Ser/Thr-linked monosaccharide that is important …

Nov 8, 2021 · In order to enable in vivo liver gene editing in HoFH patients, we developed a novel LNP delivery technology that incorporates a targeting ligand—N-acetylgalactosamine …