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Updated: Jun 24, 2025

Generation and Characterization of Human Induced Pluripotent Stem Cell-derived Astrocytes Lacking Fragile X Messenger Ribonucleoprotein
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Published on: June 6, 2025

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产生人类AMN和cALDiPSC衍生天体细胞,具有模拟X链 adrenoleukodystrophy表型的潜力

Navtej Kaur, Jaspreet Singh

    bioRxiv : the preprint server for biology
    |June 10, 2024
    PubMed
    概括

    诱导X-腺核细胞衰竭 (X-ALD) 患者的多能干细胞产生了疾病特异性星球细胞. 这些细胞积累了VLCFA,为研究X-ALD机制和测试疗法提供了一个模型.

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    Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
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