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Establishment of In Vitro FUS-Associated Familial Amyotrophic Lateral Sclerosis Model Using Human Induced Pluripotent Stem Cells
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文摘

Modeling familial ALS (FALS) using iPSC-derived motor neurons

Aberrant gene expression and/or splicing in FALS motor neuron precursor cells

Mis-localization of mutant FUS protein in FALS motor neurons

Increased apoptosis of FALS motor neurons

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