Adenine base editing rescues disrupted BCKDH function and reduces BCAAs toxic accumulation in maple syrup urine disease patient iPSC-hepatic organoids

Authors	Zhang Haiyan, Wan Zirui, Li Xiaomei, Wang Bin, Guan Jingyun, Li Yue, Jin Xiaohua, Ma Xu, Liu Guohua
Journal	Stem Cell Research & Therapy
Publication Date	2025-09-26
DOI	10.1186/s13287-025-04630-w