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 H; Pediatric Research Institute, Children's Hospital Affiliated to Shandong University (Jinan Children's Hospital), Jinan, 250022, China.; Shandong Provincial Clinical Research Center for Children's Health and Disease, Jinan, 250022, China., Wan Z; National Research Institute for Family Planning, Beijing, 100081, China.; Graduate School, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, 100005, China.; National Human Genetic Resources Center, Beijing, 102206, China., Li X; Shandong Provincial Clinical Research Center for Children's Health and Disease, Jinan, 250022, China.; Department of Neonatology, Qilu Children's Hospital of Shandong University, Jinan, 250022, Shandong, China., Wang B; Pediatric Research Institute, Children's Hospital Affiliated to Shandong University (Jinan Children's Hospital), Jinan, 250022, China.; Shandong Provincial Clinical Research Center for Children's Health and Disease, Jinan, 250022, China., Guan J; Pediatric Research Institute, Children's Hospital Affiliated to Shandong University (Jinan Children's Hospital), Jinan, 250022, China.; Shandong Provincial Clinical Research Center for Children's Health and Disease, Jinan, 250022, China., Li Y; Pediatric Research Institute, Children's Hospital Affiliated to Shandong University (Jinan Children's Hospital), Jinan, 250022, China.; Shandong Provincial Clinical Research Center for Children's Health and Disease, Jinan, 250022, China., Jin X; National Research Institute for Family Planning, Beijing, 100081, China. jinxiaohua@nrifp.org.cn.; National Human Genetic Resources Center, Beijing, 102206, China. jinxiaohua@nrifp.org.cn., Ma X; National Research Institute for Family Planning, Beijing, 100081, China. maxu_fg@nrifp.org.cn.; Graduate School, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, 100005, China. maxu_fg@nrifp.org.cn.; National Human Genetic Resources Center, Beijing, 102206, China. maxu_fg@nrifp.org.cn., Liu G; Pediatric Research Institute, Children's Hospital Affiliated to Shandong University (Jinan Children's Hospital), Jinan, 250022, China. liuguohua0204@163.com.; Shandong Provincial Clinical Research Center for Children's Health and Disease, Jinan, 250022, China. liuguohua0204@163.com. |
| Source: | Stem cell research & therapy [Stem Cell Res Ther] 2025 Sep 26; Vol. 16 (1), pp. 512. Date of Electronic Publication: 2025 Sep 26. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: BioMed Central Country of Publication: England NLM ID: 101527581 Publication Model: Electronic Cited Medium: Internet ISSN: 1757-6512 (Electronic) Linking ISSN: 17576512 NLM ISO Abbreviation: Stem Cell Res Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 1757-6512 |
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| DOI: | 10.1186/s13287-025-04630-w |
