NeurosciencebioRxivPreprint
RAPTOR-targeted Antisense Oligonucleotides Restrict Pathological mTORC1 Activity in GATOR1-dependent Disease
Imane Hadj-Aissa, Maéline Muller, Antoine Desmeules…·Paul A. Dutchak
This study develops RAPTOR-targeted ASOs to treat GATOR1-deficient diseases by reducing mTORC1 activity, improving neuronal function, and extending lifespan in models.