Ashley Summers

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.

This study develops D-siRNA, an albumin-binding dendrimer-conjugated siRNA, showing effective and durable gene silencing in the CNS with no detectable toxicity.

Therapeutic siRNAs provide safe and durable modulation of MECP2 for the treatment of mild and severe MECP2 Duplication Syndrome.

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

The paper compares somatic CAG expansion and siRNA responses in C57BL/6 and FVB BAC-CAG HD mouse models, finding comparable expansion and MSH3-targeted siRNA efficacy.

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.

This study delivers IRS2-targeting siRNAs to mammary tumors via albumin-binding dendrimers, reducing tumor growth by inhibiting Irs2 expression and altering tumor microenvironment features.

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

This study investigates how different HTT-lowering therapies affect somatic instability in HD mice, finding that transcriptional repression and CAG repeat binding reduce SI without lowering HTT levels.

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

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

The paper shows that di-valent siRNA reduces wild-type Htt mRNA but not mutant HTT mRNA in HD mouse models, with mutant transcripts remaining clustered in the nucleus.

This paper introduces a dual-targeting di-valent siRNA scaffold that can silence two genes simultaneously in the CNS of mice, demonstrating potent and programmable multi-gene modulation.

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