Swapping microglia to treat ALSP

IF 20 1区医学 Q1 NEUROSCIENCES

Nature neuroscience Pub Date : 2025-08-04 DOI:10.1038/s41593-025-02047-5

Rebecca Wright

引用次数: 0

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交换小胶质细胞治疗ALSP

成人发病伴轴突球体和色素胶质的白质脑病（ALSP）是一种由CSF1R基因突变引起的致命神经系统疾病。CSF1R主要在脑小胶质细胞中表达。在最近发表在《科学》杂志上的一项研究中，Wu等人建立了含有Csf1r突变的ALSP小鼠模型，模拟了在患者中发现的突变。这些小鼠表现出ALSP的许多表型，包括小胶质细胞数量减少，轴突球体，运动和认知缺陷。通过将表达正常CSF1R的骨髓细胞（BMCs）移植到小鼠体内，作者能够替换有缺陷的小胶质细胞，从而减轻许多病理症状。重要的是，这可以通过传统的骨髓治疗来实现。转移到临床环境，作者给8名ALSP患者注入供体bmc，并对他们进行了24个月的监测。通过代理18F-FDG PET信号测量，治疗导致患者大脑中小胶质细胞数量增加。它还减缓了脑萎缩的速度，稳定了运动和认知功能。总之，这些发现表明传统的骨髓治疗可能是治疗ALSP的一种可行的疗法。原始参考文献：Science 389， eadr1015 （2025）

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Nature neuroscience 医学-神经科学

CiteScore

38.60

自引率

1.20%

发文量

212

审稿时长

1 months

期刊介绍： Nature Neuroscience, a multidisciplinary journal, publishes papers of the utmost quality and significance across all realms of neuroscience. The editors welcome contributions spanning molecular, cellular, systems, and cognitive neuroscience, along with psychophysics, computational modeling, and nervous system disorders. While no area is off-limits, studies offering fundamental insights into nervous system function receive priority. The journal offers high visibility to both readers and authors, fostering interdisciplinary communication and accessibility to a broad audience. It maintains high standards of copy editing and production, rigorous peer review, rapid publication, and operates independently from academic societies and other vested interests. In addition to primary research, Nature Neuroscience features news and views, reviews, editorials, commentaries, perspectives, book reviews, and correspondence, aiming to serve as the voice of the global neuroscience community.