Katherine Y. Gross

One Scaffold, Many Targets: Fully Modified RNA Nanostructures for Multi-Gene Silencing Beyond the Liver

This paper introduces fully modified RNA nanostructures (FMRNs) for multi-gene silencing beyond the liver, showing enhanced pharmacokinetics, extrahepatic delivery, and programmable control over siRNA stoichiometry.

Mismatch repair dissection by in vivo RNAi reveals dose-dependent modulators of somatic instability and proteome remodeling in Huntingtons disease

The paper uses RNAi to identify dose-dependent MMR gene modulators of somatic instability and proteome changes in HD mouse models.

Divalent siRNA for prion disease

Divalent siRNA 2439-s4 lowers human PrP to 17% residual in transgenic mouse brain after one dose, and a separate mouse Prnp-targeting siRNA extends survival in prion-infected mice.

Potent and durable gene modulation in heart and muscle with chemically defined siRNAs

This study develops chemically defined siRNAs for durable gene modulation in muscle and heart, showing long-term silencing and functional benefits with minimal toxicity.