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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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 41013826 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: Adenine base editing rescues disrupted BCKDH function and reduces BCAAs toxic accumulation in maple syrup urine disease patient iPSC-hepatic organoids. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Zhang+H%22">Zhang H</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Wan+Z%22">Wan Z</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Li+X%22">Li X</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Wang+B%22">Wang B</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Guan+J%22">Guan J</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Li+Y%22">Li Y</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Jin+X%22">Jin X</searchLink>; 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.<br /><searchLink fieldCode="AU" term="%22Ma+X%22">Ma X</searchLink>; National Research Institute for Family Planning, Beijing, 100081, China. maxu&#95;fg@nrifp.org.cn.; Graduate School, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, 100005, China. maxu&#95;fg@nrifp.org.cn.; National Human Genetic Resources Center, Beijing, 102206, China. maxu&#95;fg@nrifp.org.cn.<br /><searchLink fieldCode="AU" term="%22Liu+G%22">Liu G</searchLink>; 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. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101527581%22">Stem cell research & therapy</searchLink> [Stem Cell Res Ther] 2025 Sep 26; Vol. 16 (1), pp. 512. <i>Date of Electronic Publication: </i>2025 Sep 26. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article – Name: TitleSource Label: Journal Info Group: Src Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22BioMed+Central%22">BioMed Central </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>101527581 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1757-6512 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2217576512%22">17576512 </searchLink><i>NLM ISO Abbreviation: </i>Stem Cell Res Ther <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=41013826 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1186/s13287-025-04630-w Languages: – Code: eng Text: English PhysicalDescription: Pagination: StartPage: 512 Titles: – TitleFull: Adenine base editing rescues disrupted BCKDH function and reduces BCAAs toxic accumulation in maple syrup urine disease patient iPSC-hepatic organoids. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Zhang H – PersonEntity: Name: NameFull: Wan Z – PersonEntity: Name: NameFull: Li X – PersonEntity: Name: NameFull: Wang B – PersonEntity: Name: NameFull: Guan J – PersonEntity: Name: NameFull: Li Y – PersonEntity: Name: NameFull: Jin X – PersonEntity: Name: NameFull: Ma X – PersonEntity: Name: NameFull: Liu G IsPartOfRelationships: – BibEntity: Dates: – D: 26 M: 09 Text: 2025 Sep 26 Type: published Y: 2025 Identifiers: – Type: issn-electronic Value: 1757-6512 Numbering: – Type: volume Value: 16 – Type: issue Value: 1 Titles: – TitleFull: Stem cell research & therapy Type: main |
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