fig11

RNA antisense and silencing strategies using synthetic drugs for rare muscular and neuromuscular diseases

Figure 11. Tri-GalNac chemical group addressing RNAi to the hepatocyte asialoglycoprotein receptor ASGPR. The tri-GalNac is covalently linked to the 3’ end of the siRNA sense strand, so as not to interfere with capture by RISC of the complementary antisense strand necessary for mRNA cleavage.

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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