Eric T. Wang

This study identifies disulfide bonds mediating MBNL1 and MBNL2 dimerization, showing their role in alternative splicing regulation and RNA foci formation in myotonic dystrophy.

Metformin reduces RAN proteins, improves splicing, and enhances behavioral and neuropathological outcomes in SCA8 BAC mice.

This paper introduces a novel DMD mouse model with non-uniform dystrophin expression and shows that partial dystrophin expression protects against muscle pathology.

This study shows that reducing somatic CAG expansions in HD mice improves motor function and disease markers, suggesting therapeutic potential for HD and similar repeat expansion diseases.

The paper investigates collateral RNase activity of Cas13d in mammalian cells and introduces GENO to mitigate it for repeat-targeting RNA therapies.

The paper creates a splicing atlas in the frontal cortex of DM1 patients and healthy individuals, identifying fetal-like splicing patterns and linking them to MBNL and CELF1 expression changes.

The paper analyzes RNA-seq transcriptomes from frontal cortex tissue of DM1 and DM2 patients to identify splicing changes and their implications for CNS dysfunction.

This study shows longer G4C2 repeats in C9orf72 transgenic mice lead to earlier disease onset, higher penetrance, and more RNA foci, with alternative splicing changes as early biomarkers.

This paper shows that toxic RNA repeats can catalyze the synthesis of their own inhibitors, leading to degradation and rescue of DM2-related defects.

This study shows that deactivated Cas9 reduces RNA from expanded microsatellite repeats, improving disease-related outcomes in cellular models.