Adenine base editing rescues disrupted BCKDH function and reduces BCAAs toxic accumulation in maple syrup urine disease patient iPSC-hepatic organoids
Our study provides reliable evidence that ABE8e is highly efficient and safe in correcting patient-derived HOs from MSUD, indicating the feasibility to be a transformative treatment for genetic hepatic diseases like MSUD.
Authors
- Xu Ma (ORCID: https://orcid.org/0000-0001-7674-3589)
- Xiaohua Jin (ORCID: https://orcid.org/0000-0002-8608-0755)
- Jingyun Guan
- Haiyan Zhang (ORCID: https://orcid.org/0000-0002-2877-2040)
- Guohua Liu (ORCID: https://orcid.org/0000-0002-9120-0977)
- Bin Wang (ORCID: https://orcid.org/0000-0001-9422-5750)
- Zirui Wan (ORCID: https://orcid.org/0000-0002-4721-6306)
- Xiaomei Li (ORCID: https://orcid.org/0000-0001-6505-3363)
- Yue Li (ORCID: https://orcid.org/0000-0002-6908-3591)
Institutions
- Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
- Peking Union Medical College Hospital (CN)
- Chinese National Human Genome Center (CN)
- Genetic Resources Center (JP)
- China National Petroleum and Chemical Planning Institute (CN)
- Tianjin Municipal Research Institute for Family Planning (CN)
- Qilu Hospital of Shandong University (CN)
- Shandong Center for Disease Control and Prevention (CN)
Publication Details
- Journal
- Stem Cell Research & Therapy
- Published
- 2025-09-26
- DOI
- https://doi.org/10.1186/s13287-025-04630-w
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00