Tiled base editing screens uncover rare second-site mutations that rescue a misfolded disease protein
Shivali Rawat, Parijat Sarkar, Eric Giavedoni, Ganesh V. Pusapati, Murugesh Padmanarayana, Mallory Shin, Elizabeth Phillippi, Taylar Simmons, Ruth Azaria, Diwakar Shukla, Mark L. Schultz, Rajat Rohatgi
The paper uses base editing screens to identify second-site mutations that correct misfolded NPC1 proteins, enabling therapeutic applications for diseases like Niemann-Pick type C.
bioRxiv · Nov 2, 2026 · PreprintCell Biology