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Liver-Targeted AAV-DJ-hCBS Therapy Achieves Long-Term Correction of Metabolic Imbalance in CBS-Deficient Mice.

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Title: Liver-Targeted AAV-DJ-hCBS Therapy Achieves Long-Term Correction of Metabolic Imbalance in CBS-Deficient Mice.
Authors: Joschko, Christian P.1 (AUTHOR), Wang, Chih-Chieh2 (AUTHOR), van Buiten, Azuwerus1 (AUTHOR), Goris, Maaike1,2 (AUTHOR), Hoogstra-Berends, Femke1 (AUTHOR), Wang, Joy2 (AUTHOR), Bacurio, Jan Henric2 (AUTHOR), Chen, Yinxing2 (AUTHOR), Jia, Nannan2 (AUTHOR), Deng, June2 (AUTHOR), Hu, Shiliang2 (AUTHOR), Nacht, Mariana2 (AUTHOR), Chiocco, Matthew J.2 (AUTHOR), Henning, Robert H.1 (AUTHOR), Deelman, Leo E.1 (AUTHOR) l.e.deelman@umcg.nl
Source: International Journal of Molecular Sciences. Apr2026, Vol. 27 Issue 7, p3338. 22p.
Database: Academic Search Ultimate
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ISSN:16616596
DOI:10.3390/ijms27073338