Gene therapy prevents onset of mitochondrial cardiomyopathy in neonatal mice with Ndufs6 deficiency

Gene therapy prevents onset of mitochondrial cardiomyopathy in neonatal mice with Ndufs6 deficiency

Abstract Mutations in genes affecting mitochondrial complex I (CI) can lead to mitochondrial cardiomyopathy (MCM) yet no effective treatment. This study sought to determine whether adeno-associated virus 9 (AAV9)-based gene therapy could prevent or rescue Ndufs6 deficiency-induced MCM at different d...

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Main Authors: Xiaoxian Zhang, Li Huang, Cheng Li, Jinjuan Yang, Fuyu Duan, Qiang Su, Yuelin Zhang, Meng Kou, Xiaoya Zhou, Liyan Guo, Shaoxiang Chen, Yongxia Niu, Ziyue Li, Sihua Ou, Min Zhang, Kenneth King-Yip Cheng, Jianlong Wu, Xiang Xu, Qizhou Lian
Format: Article
Language:English
Published: Nature Publishing Group 2025-05-01
Series:Cell Death Discovery
Online Access:https://doi.org/10.1038/s41420-025-02524-7
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https://doi.org/10.1038/s41420-025-02524-7

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