What is GalNAc siRNA?
What is GalNAc siRNA? GalNAc-siRNA conjugates are a simple solution to the siRNA delivery problem for liver hepatocytes and have shown the RNAi (and antisense oligonucleotide) field the path forward for targeting other tissue types. Keywords: N-acetylgalactosamine; clinical trials; liver. How does GalNAc work? GalNAc conjugates bind to the ASGPR and are taken up in endosomes, where the conjugate dissociates from the receptor. Then, the GalNAc sugars and branches are very quickly lysed from the oligonucleotide before the oligonucleotide escapes to the cytoplasm by a still poorly understood mechanism. How does RNA interference regulate gene expression? RNAi is short for...