Raymond C. Furgal

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.

High-throughput quantification of huntingtin mRNA expression and aggregation in mouse brain using automated RNAscope imaging.

This study quantifies huntingtin mRNA expression and aggregation in mouse brains using a high-throughput RNAscope pipeline, revealing heterogeneity in mRNA accumulation and localization across brain regions.

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.

Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntingtons disease pathogenesis in mice

This study shows that combining RNAi to silence MSH3 and HTT reduces huntingtin inclusions and restores gene expression in HD mice.

RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice

The paper shows that silencing SOD1 with optimized di-siRNA extends survival and prevents disease progression in ALS mice.

Di-valent siRNA Mediated Silencing of MSH3 Blocks Somatic Repeat Expansion in Mouse Models of Huntington's Disease

Silencing MSH3 in the CNS of two models of Huntington's disease using di-valent siRNA blocks disease-accelerating somatic expansion of CAG repeats.