Ryan J. Marina

This paper generates monoclonal antibodies and intrabodies that selectively target misfolded TDP-43 aggregates in disease, showing they inhibit transmission and promote degradation in cell models.

The paper identifies that transcriptional repression via BET inhibition and upregulation of NRMKS genes maintains pluripotency in diapause ES cells by suppressing MAP kinase signaling.

This study develops a CRISPR-Cas13d system targeting CAG-expanded HTT RNA in HD models, reducing mutant mRNA and reversing disease phenotypes without significant off-target effects.