研究结果为DMD发病机制，特别是疾病早期的分子和细胞变化提供了新的见解，揭示了免疫、纤维化以及肌肉干细胞在DMD早期的动态变化，为早期干预和靶向治疗提供了科学依据。此外，DMD猴模型显示FAPs纤维化不依赖于TGFβ通路，为新药研发提供了新的方向。更重要的是，肌肉干细胞功能缺陷导致了肌肉修复障碍，提示DMD是一种干细胞疾病，开发细胞治疗或针对肌肉干细胞进行干预治疗也许是未来研究的重要方向。

About Solid Biosciences Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene ...

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An important subgroup are the dystrophin-related muscular dystrophies caused by variants in the DMD gene. They can be divided into the more severe Duchenne muscular dystrophy and the milder Becker ...

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The Billings Livestock Commission hosted the sixth annual Calves to Cure to help raise money and awareness for Duchenne ...