NeurosciencebioRxivPreprint
Domain-specific regulation of PABPN1 uncouples aggregation from function and enables therapeutic rescue
Milad Shademan, Iolanda Vendrell, Alberto Malerba…·Vered Raz
This study identifies a stabilized PABPN1 variant, trPAB, that reduces aggregation and restores function in OPMD and bladder cancer models, supporting its potential as a gene therapy.
DOI 10.64898/2026.06.12.731084