{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T21:16:30Z","timestamp":1784841390374,"version":"3.55.0"},"reference-count":57,"publisher":"Oxford University Press (OUP)","issue":"5","license":[{"start":{"date-parts":[[2026,2,25]],"date-time":"2026-02-25T00:00:00Z","timestamp":1771977600000},"content-version":"vor","delay-in-days":1,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["32370853"],"award-info":[{"award-number":["32370853"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,2,24]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Oligodendrocytes (OLs), the myelinating cells of the central nervous system, are particularly prone to pathogenic G-to-A mutations, such as PLP1A243V, which causes Pelizaeus\u2013Merzbacher disease (PMD), a lethal hypomyelinating disorder lacking effective therapy. Although adenine base editors (ABEs) can in principle correct such mutations, their application in OLs is limited by inefficient on-target editing. Here, we develop a spatially concentrated ABE (cABE) strategy that enhances editing by promoting nuclear translocation of tRNA adenosine deaminase (TadA*) from the cytoplasm. Using a SunTag-based multivalent recruitment system, TadA* is locally enriched at genomic targets (cABE-1.0), achieving robust editing in vitro. To enable in vivo delivery while preserving high efficiency and fidelity, SpCas9 is replaced with compact eNme2-C Cas9, generating an AAV-compatible variant (cABE-2.0). Notably, cABE-2.0 forms dynamic nuclear puncta with properties of liquid\u2013liquid phase separation, enhancing on-target editing while substantially reducing transcriptome-wide RNA off-target effects. Functionally, cABE-2.0 efficiently corrects the PLP1A243V mutation in OLs, restores Plp subcellular localization, and rescues myelination-related phenotypes. These findings demonstrate that spatial reorganization, rather than increasing intrinsic catalytic activity of TadA*, provides a distinct principle for improving base editing in difficult-to-edit cell types, such as OLs, offering a mechanistic and technical framework for gene therapy of PMD and related myelin disorders.<\/jats:p>","DOI":"10.1093\/nar\/gkag156","type":"journal-article","created":{"date-parts":[[2026,2,25]],"date-time":"2026-02-25T07:24:25Z","timestamp":1772004265000},"source":"Crossref","is-referenced-by-count":1,"title":["Spatially concentrated adenine base editors efficiently correct\n                    <i>PLP1<\/i>\n                    mutations in oligodendrocytes"],"prefix":"10.1093","volume":"54","author":[{"given":"Chi","family":"Zhang","sequence":"first","affiliation":[{"name":"Department of Histoembryology, Genetics and Developmental Biology, Shanghai Key Laboratory of Reproductive Medicine, Shanghai Jiao Tong University School of Medicine , Shanghai 200025 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Developmental Biology, Shanghai Key Laboratory of Reproductive Medicine, Shanghai Jiao Tong University School of Medicine , Shanghai 200025 ,","place":["China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pingping","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Histoembryology, Genetics and Developmental Biology, Shanghai Key Laboratory of Reproductive Medicine, Shanghai Jiao Tong University School of Medicine , Shanghai 200025 ,","place":["China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xinyue","family":"Jiang","sequence":"additional","affiliation":[{"name":"Department of Histoembryology, Genetics and Developmental Biology, Shanghai Key Laboratory of Reproductive Medicine, Shanghai Jiao Tong University School of Medicine , Shanghai 200025 ,","place":["China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cuiping","family":"Yang","sequence":"additional","affiliation":[{"name":"The International Peace Maternity and Child Health Hospital, Shanghai Key Laboratory of Embryo Original Diseases, Shanghai Jiao Tong University School of Medicine , Shanghai 200030 ,","place":["China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aibin","family":"Liang","sequence":"additional","affiliation":[{"name":"Department of Hematology, Tongji Hospital, Tongji University School of Medicine , Shanghai 200065 ,","place":["China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6558-791X","authenticated-orcid":false,"given":"Jian","family":"Zhang","sequence":"additional","affiliation":[{"name":"Key Laboratory of Protection, Development and Utilization of Medicinal Resources in Liupanshan Area, Ministry of Education, Peptide & Protein Drug Research Center, College of Pharmacy, Ningxia Medical University , Yinchuan, Ningxia 750004 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Embryo Original Diseases, Shanghai Jiao Tong University School of Medicine , Shanghai 200030 ,","place":["China"]},{"name":"Department of Histology and Embryology, Zunyi Medical University , Zunyi, Guizhou 563000 ,","place":["China"]},{"name":"School of Basic Medical Sciences, Ningxia Medical University , Yinchuan, Ningxia 750004 ,","place":["China"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2026,2,25]]},"reference":[{"key":"2026022502241723400_B1","doi-asserted-by":"publisher","first-page":"1955","DOI":"10.1016\/j.cell.2024.02.025","article-title":"Contrasting somatic mutation patterns in aging human neurons and oligodendrocytes","volume":"187","author":"Ganz","year":"2024","journal-title":"Cell"},{"key":"2026022502241723400_B2","doi-asserted-by":"publisher","first-page":"239","DOI":"10.1146\/annurev-genom-121520-081242","article-title":"Brain somatic mutation in aging and Alzheimer\u2019s 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