A scoping review of stem cell models of leukodystrophies: advances in understanding pathophysiological mechanisms
6.5
来源：
Nature
关键字：
neural coding
发布时间：
2025-11-28 23:59
摘要：
This scoping review examines the advancements in stem cell models for leukodystrophies, a group of rare genetic disorders affecting the central nervous system. It discusses the challenges and future directions in utilizing these models to enhance understanding of disease mechanisms and aid in therapeutic development. The review emphasizes the potential of induced pluripotent stem cells (iPSCs) in generating patient-specific models, which are crucial for studying the pathophysiology of leukodystrophies and developing effective treatments.
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1.5分
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关键证据
Stem cells have emerged as a transformative tool in leukodystrophy research.
The review highlights recent developments, challenges, and future directions in leveraging these models.
iPSCs provide an alternative by reprogramming somatic cells into a pluripotent state.
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AI评分总结
This scoping review examines the advancements in stem cell models for leukodystrophies, a group of rare genetic disorders affecting the central nervous system. It discusses the challenges and future directions in utilizing these models to enhance understanding of disease mechanisms and aid in therapeutic development. The review emphasizes the potential of induced pluripotent stem cells (iPSCs) in generating patient-specific models, which are crucial for studying the pathophysiology of leukodystrophies and developing effective treatments.