Correction of a recurrent pathogenic variant in methylmalonic acidemia using adenine base editing
Elena Kahn, Hooda Said, Ping Qü, Mohamad‐Gabriel Alameh, Xiao Wang, Kiran Musunuru, Rebecca C. Ahrens‐Nicklas
This study corrects a common pathogenic variant in MMA using adenine base editing in hepatocytes, showing efficient in vitro editing with minimal off-target effects.
bioRxiv · Mar 15, 2026 · PreprintGenetics