Fernando G. Vieira

The paper generates and validates a human iPSC-derived TDP-43 knockout model for ALS, showing widespread cryptic exon inclusion and identifying potential therapeutic modulators.

This study evaluates three PERK modulators in an ALS mouse model and finds they do not provide therapeutic benefits, with some effects possibly mediated through PERK-independent mechanisms.

The paper identifies Type I PRMT inhibitors as potential therapeutics for polyGR- and polyPR-related toxicity by showing ADMe of polyGR is critical for its pathogenesis.