From bench to bedside: Improving the clinical safety of GalNAc–siRNA conjugates using seed-pairing destabilization
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DOI: 10.1093/nar/gkac539
Abstract: Abstract Preclinical mechanistic studies have pointed towards RNA interference-mediated off-target effects as a major driver of hepatotoxicity for GalNAc–siRNA conjugates. Here, we demonstrate that a single glycol nucleic acid or 2′–5′-RNA modification can substantially reduce… read more here.
Keywords: safety; seed pairing; sirna conjugates; galnac sirna ... See more keywords