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Nature:小胶质细胞替换疗法,治疗致命大脑疾病
生物世界· 2025-08-18 04:05
Core Viewpoint - The research presents a novel allogeneic brain microglia replacement therapy that does not require myeloablation, showing significant potential for treating lysosomal storage disorders and improving patient outcomes [3][4][7]. Group 1: Research Findings - The study developed a brain-specific, efficient microglia replacement therapy that effectively treated a mouse model of lysosomal storage disease, nearly doubling their lifespan and restoring motor coordination [4][7]. - The research revealed that hematopoietic stem cells are not necessary for reconstructing the brain's myeloid compartment, as Sca1+ progenitor cells can efficiently replace microglia without the need for systemic myeloablation [7][8]. - In the Sandhoff disease mouse model, over 85% of microglia were replaced by the injected Sca1+ progenitor cells, leading to significant survival improvements, with some mice living up to 250 days compared to an average of 135 days for untreated mice [7][8]. Group 2: Implications for Future Treatments - The findings suggest a pathway for developing allogeneic microglia therapies for brain diseases, overcoming the limitations of traditional hematopoietic stem cell transplantation [8]. - The study also demonstrated that human-induced pluripotent stem cell-derived myeloid progenitor cells exhibit similar implantation potential, indicating cross-species conservation of this therapeutic approach [8]. - Another related study highlighted the role of microglia in maintaining brain homeostasis and the potential for therapeutic interventions in neurodegenerative diseases like Sandhoff disease [11].