Friedreich Ataxia iPSC-derived microglia display an interferon-responsive, antigen-presenting, and inflammasome-associated state with lysosomal biology and extracellular matrix abnormalities
本研究表明,来自弗里德赖希共济失调患者的诱导多能干细胞(iPSC)来源的小胶质细胞表现出内在的、细胞自主性的功能障碍,其特征为干扰素反应性、抗原提呈性和促炎状态,并伴随溶酶体、自噬和细胞外基质异常,从而确定了作为该疾病潜在治疗靶点的特定分子通路。