BiochemistrybioRxiv
Ras-dependent RAF-MAPK hyperactivation by pathogenic RIT1 is a therapeutic target in Noonan syndrome-associated cardiac hypertrophy
A. Cuevas-Navarro, M. Wagner, R. Van…·P. Castel
This study identifies how pathogenic RIT1 mutations drive RAF-MAPK hyperactivation in Noonan syndrome, showing MEK inhibition can alleviate cardiac hypertrophy in a mouse model.
DOI 10.1126/sciadv.adf4766