Ataxia-Telangiectasia: An emerging role for inflammation in driving neurodegeneration and premature ageing

共济失调-毛细血管扩张症（A-T）是一种毁灭性的遗传性疾病，发生在儿童早期，导致患者在二十多岁时死亡。到目前为止，还没有治愈的方法，迫切需要治疗方法。该项目将使用来自A-T患者干细胞的最先进的脑类器官，以更好地了解这种疾病，并评估针对驱动A-T儿童慢性炎症和脑神经退行性变的分子机制的新药。

Ataxia-Telangiectasia (A-T) is a devastating genetic disease that arises in early childhood and causes patients to die in their twenties. To date there is no cure, and therapeutics are desperately needed. This project will use state-of-the-art brain organoids derived from stem cells of A-T patients in order to better understand this disease and evaluate novel drugs that target the molecular mechanisms that drive chronic inflammation and brain neurodegeneration in children with A-T.