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脑内“换细胞”让患病动物寿命翻倍
Huan Qiu Wang Zi Xun· 2025-08-11 01:45
Core Insights - A groundbreaking study published in Nature demonstrates the use of non-genetically matched healthy precursor cells to replace over half of the diseased microglia in Sandhoff disease mice, extending their lifespan from 135 days to 250 days and restoring motor functions and exploratory behavior to near-normal levels [1][2] Group 1: Research Findings - The study provides a blueprint for "off-the-shelf" cell therapy for currently untreatable neurodegenerative diseases like Tay-Sachs and Sandhoff diseases, which are lysosomal storage disorders characterized by rapid degeneration and early mortality in affected children [1] - The research team employed a "brain-region-specific transplantation" strategy, using low-dose radiation and drugs to temporarily clear existing microglia in the mice's brains before injecting microglial precursor cells from non-matching donors [1][2] - The new cells maintained over 85% of the total microglial cell population in the brain after 8 months and did not spread to other body parts, indicating a successful integration [2] Group 2: Implications for Future Treatments - The approach addresses three major challenges: it does not require systemic toxic preconditioning, avoids gene editing to supplement missing enzymes, and prevents rejection reactions [2] - The components used in the therapy, including radiation doses, microglial-clearing agents, and immunosuppressants, are already approved for other diseases, suggesting a potential for rapid clinical application [2] - The research indicates that similar microglial dysfunctions are present in common neurodegenerative diseases like Alzheimer's and Parkinson's, which could benefit from this therapy if human trials are successful [2]