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Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference

18 Apr 2023

Abstract excerpt

Herein, we report the systematic investigation of stereopure phosphorothioate (PS) and phosphoryl guanidine (PN) linkages on siRNA-mediated silencing. The incorporation of appropriately positioned and configured stereopure PS and PN linkages to N-acetylgalactosamine (GalNAc)-conjugated siRNAs based on multiple targets (Ttr and HSD17B13) increased potency and durability of mRNA silencing in mouse hepatocytes in...

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Share to Advanced biosensing and bioanalysis techniques
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PMID: 37070173
DOI: 10.1093/nar/gkad268

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