An oversized AAV8 vector to deliver CPS1

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Li2025Oversized-CCBYNCND.pdf (1.66 MB)
Date
2025-03-19
Authors
Li, Shuang
Zhang, Chen
Han, Renzhi
Language
American English
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Pediatrics, School of Medicine
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Elsevier
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Carbamoyl phosphate synthetase 1 (CPS1) deficiency, Urea cycle disorders, Hyperammonemia, Coma, Death
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Cite As
Li 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
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Molecular Therapy - Nucleic Acids
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Attribution-NonCommercial-NoDerivatives 4.0 International Creative Commons licenses
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PMC
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Article
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https://hdl.handle.net/1805/48072
DOI
https://doi.org/10.1016/j.omtn.2025.102504
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Final published version
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