GeneticsbioRxivPreprint
Correction of a recurrent pathogenic variant in methylmalonic acidemia using adenine base editing
Elena Kahn, Hooda Said, Ping Qü…·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.
DOI 10.64898/2026.03.12.711365