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生产C9orf72重复敲进iPSC线路用于模拟ALS和FTD

Rachel Coneys1,2, Alexander J Cammack1,2, Remya R Nair3

  • 1UK Dementia Research Institute at UCL, London, UK.

bioRxiv : the preprint server for biology
|October 1, 2025
PubMed
概括

诱导多能干细胞 (iPSC) 模型用于肌缩侧硬化症 (ALS) 和前性痴呆症 (FTD) 的研究,使用单一参考线进行了标准化. 基因编辑引入了C9orf72突变,创造了具有特征病理学的疾病模型.

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