GalNAc
Tris GalNAc (N-acetylgalactosamine) with the optimized linker structure can be introduced in RNA and DNA to improve receptor-mediated delivery to hepatocytes via ASialoGlycoProtein Receptor (ASGPR). This receptor is exclusively presented in hepatocytes in the liver that drives efficient and selective delivery in vivo. Conjugation of ASO and siRNA with GalNAc is widely used for the targeted delivery into freshly isolated hepatocytes and in vivo.
Available sites for modifications are 3’.
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