Article
Use of a Transgenic Human PNPLA3I148M Knock-in Mouse for Translational Safety Evaluations of siRNA Therapeutics.
Nucleic acid therapeutics - 1 Dec 2025
Brooks Ben, Shkumatov Artem, Kalanzi Jackson, Henderson Park Kim, Lade Julie M, Wong Diana, Dou Hui, Long Jason, Rulifson Ingrid C, Murray Justin K, Mihalcik Lauren, Harper Tod A
Abstract excerpt
The PNPLA3 single nucleotide polymorphism, rs738409, is the strongest known genetic risk factor for metabolic dysfunction-associated steatotic liver disease; thus, targeting the minor allele with a GalNAc-conjugated siRNA is an attractive strategy to treat patients carrying the genetic variant. To enable translational safety assessment of a GalNAc-conjugated siRNA that specifically targets the rs738409 sequence...
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