Kathryn R. Monopoli

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

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

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

The paper develops a machine learning framework for predicting siRNA efficacy using a small dataset by applying trichotomous partitioning and random forest models, achieving better performance than linear models.