In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression.
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| Title: | In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression. |
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| Authors: | Nam BG; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Medical Research Center of Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea.; Graduate Program of Biomedical Engineering, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea., Seo JH; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Department of Biohealth Engineering, Graduate school of Transdisciplinary Health Sciences, Yonsei University, Seoul, 03722, Republic of Korea., Hong SA; Medical Research Center of Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea., Kim JH; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea., Kang M; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea., Notario GR; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea., Hong Y; AAVATAR Therapeutics, 12925, Gyeonggi-do, Republic of Korea., Cho S; AAVATAR Therapeutics, 12925, Gyeonggi-do, Republic of Korea., Bae S; Medical Research Center of Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea. sbae7@snu.ac.kr.; Department of Biomedical Sciences, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea. sbae7@snu.ac.kr.; Cancer Research Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea. sbae7@snu.ac.kr., Cho SR; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Graduate Program of Biomedical Engineering, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Department of Biohealth Engineering, Graduate school of Transdisciplinary Health Sciences, Yonsei University, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Rehabilitation Institute of Neuromuscular Disease, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Brain Research Institute, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Metabolism-Dementia Research Institute, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac. |
| Source: | Genome medicine [Genome Med] 2026 Apr 23; Vol. 18 (1). Date of Electronic Publication: 2026 Apr 23. |
| Publication Type: | Journal Article; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: BioMed Central Country of Publication: England NLM ID: 101475844 Publication Model: Electronic Cited Medium: Internet ISSN: 1756-994X (Electronic) Linking ISSN: 1756994X NLM ISO Abbreviation: Genome Med Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| FullText | Text: Availability: 0 |
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| Header | DbId: mdl DbLabel: MEDLINE Ultimate An: 42026681 AccessLevel: 2 PubType: Academic Journal PubTypeId: academicJournal PreciseRelevancyScore: 0 |
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| Items | – Name: Title Label: Title Group: Ti Data: In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression. – Name: Author Label: Authors Group: Au Data: <searchLink fieldCode="AU" term="%22Nam+BG%22">Nam BG</searchLink>; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Medical Research Center of Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea.; Graduate Program of Biomedical Engineering, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Seo+JH%22">Seo JH</searchLink>; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Department of Biohealth Engineering, Graduate school of Transdisciplinary Health Sciences, Yonsei University, Seoul, 03722, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Hong+SA%22">Hong SA</searchLink>; Medical Research Center of Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Kim+JH%22">Kim JH</searchLink>; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Kang+M%22">Kang M</searchLink>; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Notario+GR%22">Notario GR</searchLink>; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Hong+Y%22">Hong Y</searchLink>; AAVATAR Therapeutics, 12925, Gyeonggi-do, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Cho+S%22">Cho S</searchLink>; AAVATAR Therapeutics, 12925, Gyeonggi-do, Republic of Korea.<br /><searchLink fieldCode="AU" term="%22Bae+S%22">Bae S</searchLink>; Medical Research Center of Genomic Medicine Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea. sbae7@snu.ac.kr.; Department of Biomedical Sciences, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea. sbae7@snu.ac.kr.; Cancer Research Institute, Seoul National University College of Medicine, Seoul, 03080, Republic of Korea. sbae7@snu.ac.kr.<br /><searchLink fieldCode="AU" term="%22Cho+SR%22">Cho SR</searchLink>; Department and Research Institute of Rehabilitation Medicine, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Graduate Program of Biomedical Engineering, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Department of Biohealth Engineering, Graduate school of Transdisciplinary Health Sciences, Yonsei University, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Graduate School of Medical Science, Brain Korea 21 Project, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Rehabilitation Institute of Neuromuscular Disease, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Brain Research Institute, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac.; Metabolism-Dementia Research Institute, Yonsei University College of Medicine, Seoul, 03722, Republic of Korea. srcho918@yuhs.ac. – Name: TitleSource Label: Source Group: Src Data: <searchLink fieldCode="JN" term="%22101475844%22">Genome medicine</searchLink> [Genome Med] 2026 Apr 23; Vol. 18 (1). <i>Date of Electronic Publication: </i>2026 Apr 23. – Name: TypePub Label: Publication Type Group: TypPub Data: Journal Article; Research Support, Non-U.S. Gov't – 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>101475844 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1756-994X (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%221756994X%22">1756994X </searchLink><i>NLM ISO Abbreviation: </i>Genome Med <i>Subsets: </i>MEDLINE |
| PLink | https://search.ebscohost.com/login.aspx?direct=true&site=eds-live&db=mdl&AN=42026681 |
| RecordInfo | BibRecord: BibEntity: Identifiers: – Type: doi Value: 10.1186/s13073-026-01620-2 Languages: – Code: eng Text: English Titles: – TitleFull: In vivo adenine base editing of mutant Galc gene ameliorates Krabbe disease progression. Type: main BibRelationships: HasContributorRelationships: – PersonEntity: Name: NameFull: Nam BG – PersonEntity: Name: NameFull: Seo JH – PersonEntity: Name: NameFull: Hong SA – PersonEntity: Name: NameFull: Kim JH – PersonEntity: Name: NameFull: Kang M – PersonEntity: Name: NameFull: Notario GR – PersonEntity: Name: NameFull: Hong Y – PersonEntity: Name: NameFull: Cho S – PersonEntity: Name: NameFull: Bae S – PersonEntity: Name: NameFull: Cho SR IsPartOfRelationships: – BibEntity: Dates: – D: 23 M: 04 Text: 2026 Apr 23 Type: published Y: 2026 Identifiers: – Type: issn-electronic Value: 1756-994X Numbering: – Type: volume Value: 18 – Type: issue Value: 1 Titles: – TitleFull: Genome medicine Type: main |
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