Rula Zain

This paper investigates how (AAGGG)n repeats in the RFC1 gene cause DNA replication fork stalling and reversal, implicating G4-DNA and SMARCAL1 in the process.

This study shows that chemical modifications to siRNA and linkers in AOCs can improve potency and tissue targeting while allowing higher payload loading.

This study compares lipid-conjugated ASO delivery in mice, showing differential tissue and CNS targeting based on lipid type and administration route.

This study develops a click chemistry-based strategy for siRNA conjugation to enhance extrahepatic RNAi, finding that bis-fatty-acid conjugates improve activity in heart and muscle while showing modality-specific limitations.

This study shows that anti-gene oligonucleotides targeting GAA*TTC repeats in Friedreichs ataxia increase FXN expression in patient cells.

This study develops an experimental system to analyze GAA repeat expansions and replication fork stalling in human cells, finding that repeat expansions depend on proteins involved in replication fork stalling and restart.

This study shows that LNA-DNA and PNA oligomers prevent GAA·TTC repeat expansions in human cells by disrupting triplex DNA structures formed by these repeats.