Nair, Jayaprakash K. published the artcileMultivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing, Application In Synthesis of 10378-06-0, the publication is Journal of the American Chemical Society (2014), 136(49), 16958-16961, database is CAplus and MEDLINE.
Conjugation of small interfering RNA (siRNA) to an asialoglycoprotein receptor ligand derived from N-acetylgalactosamine (GalNAc) facilitates targeted delivery of the siRNA to hepatocytes in vitro and in vivo. The ligands derived from GalNAc are compatible with solid-phase oligonucleotide synthesis and deprotection conditions, with synthesis yields comparable to those of standard oligonucleotides. S.c. (SC) administration of siRNA-GalNAc conjugates resulted in robust RNAi-mediated gene silencing in liver. Refinement of the siRNA chem. achieved a 5-fold improvement in efficacy over the parent design in vivo with a median ED (ED50) of 1 mg/kg following a single dose. This enabled the SC administration of siRNA-GalNAc conjugates at therapeutically relevant doses and, importantly, at dose volumes of ≤1 mL. Chronic weekly dosing resulted in sustained dose-dependent gene silencing for over 9 mo with no adverse effects in rodents. The optimally chem. modified siRNA-GalNAc conjugates are hepatotropic and long-acting and have the potential to treat a wide range of diseases involving liver-expressed genes.
Journal of the American Chemical Society published new progress about 10378-06-0. 10378-06-0 belongs to esters-buliding-blocks, auxiliary class Other Aliphatic Heterocyclic,Chiral,Ester,Inhibitor,Inhibitor, name is (3aR,5R,6R,7R,7aR)-5-(Acetoxymethyl)-2-methyl-5,6,7,7a-tetrahydro-3aH-pyrano[3,2-d]oxazole-6,7-diyl diacetate, and the molecular formula is C14H19NO8, Application In Synthesis of 10378-06-0.
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