Author

Brianna Bramato

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Open access Oct 2026

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

Therapeutic siRNAs are a major clinical modality for hepatic gene silencing, but efficient extrahepatic delivery and multi-gene modulation remain significant barriers. Nucleic acid nanotechnology offers programmable assembly, but conventional structures suffer from enzymatic instability in vivo. Here we report fully ch...

H. Fakih, Ashley Summers, Qi Tang et al. · bioRxiv · 0 citations

Aug 2026

Blocking somatic repeat expansion and lowering huntingtin by RNAi synergize to attenuate Huntington's disease pathogenesis in mice.

These findings position somatic expansion as a promising therapeutic target and demonstrate the potential of RNAi-based cosilencing of MSH3 and HTT as a disease-modifying strategy for HD.

Jillian Belgrad, Ashley Summers, C. Landles et al. · Science Translational Medici... · 0 citations