enOsCas12f1-mediated exon skipping for Duchenne muscular dystrophy therapy in humanized mouse model

Ming Jin; Jiajia Lin; Yu Zhang; Qingquan Xiao; Xiangfeng Kong; Xiumei Zhang; Zhurui Shao; Yin Wang; Yuyang Yu; Jinjing Li; Wan-Jin Chen; Guoling Li; Hui Yang; Ning Wang

doi:10.1016/j.jgg.2023.12.003

Volume 51 Issue 2

Feb. 2024

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Article Navigation > Journal of Genetics and Genomics > 2024 > 51(2): 256-259

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enOsCas12f1-mediated exon skipping for Duchenne muscular dystrophy therapy in humanized mouse model

doi: 10.1016/j.jgg.2023.12.003

1. Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, Fujian 350004, China;
2. HUIDAGENE Therapeutics Co., Ltd., Shanghai 200131, China;
3. Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, Fujian 350004, China;
4. HUIDAGENE Therapeutics Co., Ltd., Shanghai 200131, China;
5. HUIDAGENE Therapeutics Co., Ltd., Shanghai 200131, China Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai 200031, China;
6. Department of Neurology and Institute of Neurology of First Affiliated Hospital, Institute of Neuroscience, Fujian Key Laboratory of Molecular Neurology, Fujian Medical University, Fuzhou, Fujian 350004, China

Funds:

This work was supported by grants from National Natural Science Foundation of China (82202052, 82230039, U2005201, 81870902, 82001217, 31925016, and 82021001), Major Scientific Research Project for Middle-age and Youth of Fujian Provincial Health Commission grant (2021ZQNZD003), Major Scientific Research Project of Fujian Provincial Health Commission grant (2022ZD01002), Joint Funds for the Innovation of Science and Technology of Fujian Province (2018Y9082), and the Youth Scientific Research Project of Fujian Provincial Health Commission (2020QNA045).

Received Date: 2023-05-14
Accepted Date: 2023-12-09
Rev Recd Date: 2023-12-08

Available Online: 2025-06-07

Publish Date: 2023-12-14

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References(17)

References

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