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.
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
Description
ISSN:1756-994X
DOI:10.1186/s13073-026-01620-2