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针对PLP1的反意义寡核酸改善FOXG1综合征的小鼠

Daniel C S Tan1, Seonghee Jung1, Yuanyuan Deng1

  • 1Dementia Research Centre, Department of Biomedical Sciences, Faculty of Medicine Health and Human Sciences, Macquarie University, Sydney, NSW 2109, Australia.

International journal of molecular sciences
|October 16, 2024
PubMed
概括

罕见的神经发育障碍FOXG1综合征,在新疗法方面表现有前途. 向蛋白质脂蛋白1 (PLP1) 与反感性寡核酸 (ASOs) 改善了小鼠模型中的神经系统缺陷.

关键词:
这是FOXG1综合征.无意义的寡核酸 (ASO) 是一种反意义的寡核酸 (ASO).基因治疗的基因疗法鼠标模型 鼠标模型鼠标模型神经发育障碍是一种神经发育障碍.蛋白质脂蛋白1 (PLP1) 是一种蛋白质脂蛋白.

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