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中国专家创新治疗范式 推动“细胞修复大脑”走向更多临床场景
Huan Qiu Wang Zi Xun· 2025-07-11 06:38
Core Insights - The research published in "Science" confirms that replacing pathogenic microglia in the central nervous system can successfully block adult-onset leukodystrophy (ALSP), a condition with no effective treatment options available globally [1][2] - The study represents a full chain from genetic mechanisms to clinical validation, marking a new phase in brain disease cell repair and potentially expanding to other neurological disorders [1][2] Group 1: Research Findings - The research team achieved over 90% efficient replacement of microglia in animal models and completed two years of follow-up with multiple ALSP patients, demonstrating that traditional bone marrow cell transplantation (tBMT) can innovate mechanisms to replace microglia and prevent disease progression [1][2] - ALSP is a hereditary neurodegenerative disease with an average lifespan of only 3 to 6.8 years post-onset, caused by genetic mutations leading to dysfunctional microglia that degrade cognitive and motor functions [2] Group 2: Future Implications - The study establishes a treatment paradigm of "gene mutation-cell function abnormality-precise replacement," providing hope for ALSP patients and aiming to extend the technology to other neurological diseases characterized by microglial dysfunction [3] - The research team has publicly shared the microglial replacement operational protocol globally, with plans to promote the application of this technology in more clinical scenarios [3]