这篇文章概述了一项关于小胶质细胞替代疗法治疗CSF1R相关性小胶质细胞病(ALSP)的研究。该研究首先通过创建模仿人类热点突变的小鼠模型,证明了小胶质细胞替代(MISTER)策略能有效纠正致病性CSF1R突变,从而改善小鼠的脑部病理、运动和认知功能障碍。此外,研究还发现传统骨髓移植(tBMT)在ALSP小鼠和患者体内能有效替代小胶质细胞,并展现出类似的治疗益处。最后,研究通过对八名ALSP患者进行tBMT治疗的临床验证,观察到疾病进展停止,运动和认知功能得到维持。References: Jingying Wu et al. Microglia replacement halts the progression of microgliopathy in mice and humans.Science389,eadr1015(2025).DOI:10.1126/science.adr1015
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