Adenine base editing rescues disrupted BCKDH function and reduces BCAAs toxic accumulation in maple syrup urine disease patient iPSC-hepatic organoids.
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| Title: | Adenine base editing rescues disrupted BCKDH function and reduces BCAAs toxic accumulation in maple syrup urine disease patient iPSC-hepatic organoids. |
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| Authors: | Zhang, Haiyan1,2 (AUTHOR), Wan, Zirui3,4,5 (AUTHOR), Li, Xiaomei2,6 (AUTHOR), Wang, Bin1,2 (AUTHOR), Guan, Jingyun1,2 (AUTHOR), Li, Yue1,2 (AUTHOR), Jin, Xiaohua3,5 (AUTHOR) jinxiaohua@nrifp.org.cn, Ma, Xu3,4,5 (AUTHOR) maxu_fg@nrifp.org.cn, Liu, Guohua1,2 (AUTHOR) liuguohua0204@163.com |
| Source: | Stem Cell Research & Therapy. 9/26/2025, Vol. 16 Issue 1, p1-17. 17p. |
| Database: | Academic Search Ultimate |
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| ISSN: | 17576512 |
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| DOI: | 10.1186/s13287-025-04630-w |
