An oversized AAV8 vector to deliver CPS1

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dc.contributor.authorLi, Shuang
dc.contributor.authorZhang, Chen
dc.contributor.authorHan, Renzhi
dc.contributor.departmentPediatrics, School of Medicine
dc.date.accessioned2025-05-14T06:49:22Z
dc.date.available2025-05-14T06:49:22Z
dc.date.issued2025-03-19
dc.eprint.versionFinal published version
dc.identifier.citationLi S, Zhang C, Han R. An oversized AAV8 vector to deliver CPS1. Mol Ther Nucleic Acids. 2025;36(2):102504. Published 2025 Mar 19. doi:10.1016/j.omtn.2025.102504
dc.identifier.urihttps://hdl.handle.net/1805/48072
dc.language.isoen_US
dc.publisherElsevier
dc.relation.isversionof10.1016/j.omtn.2025.102504
dc.relation.journalMolecular Therapy - Nucleic Acids
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 Internationalen
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.sourcePMC
dc.subjectCarbamoyl phosphate synthetase 1 (CPS1) deficiency
dc.subjectUrea cycle disorders
dc.subjectHyperammonemia
dc.subjectComa
dc.subjectDeath
dc.titleAn oversized AAV8 vector to deliver CPS1
dc.typeArticle
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