Modulating Fibro/Adipogenic Precursors to Prevent Adipogenic Replacement of Muscle

抽象的。 成功的肌肉再生需要复杂的细胞编排， 去除和替换受损纤维的细胞群。这种编排的中断导致了 肌肉变性和肌纤维的纤维性或成脂性替代。这种现象是 尤其流行于肌营养不良症，进行性肌肉丧失导致严重的 虚弱和早期的生命损失。因此，人们越来越需要了解细胞 有助于肌纤维再生的种群及其相互作用，但可能导致 当出现问题时，进行纤维性或成脂性置换。本项目旨在了解 细胞相互作用受损导致肌纤维丢失和肌肉损伤的机制 2B型肢带型肌营养不良症的虚弱性及其预防的测试方法 减损。这一理解将有助于开发新的靶向治疗方法 阻止本病和其他目前无症状的肌营养不良的疾病进展 有效的治疗选择。

ABSTRACT. Successful muscle regeneration requires an elaborate cellular choreography between multiple cell populations to remove and replace damaged fibers. Disruption of this choreography leads to muscle degeneration and fibrotic or adipogenic replacement of myofibers. This phenomenon is particularly prevalent in muscular dystrophies, where progressive muscle loss leads to severe debilitation and early loss of life. Hence, there is growing need to understand the cell populations and their interactions which contribute to myofiber regeneration, but can lead to fibrotic or adipogenic replacement when gone awry. This project aims to understand the mechanism underlying impaired cellular interactions leading to myofiber loss and muscle weakness in limb girdle muscular dystrophy type 2B and test approaches to prevent this impairment. This understanding will help develop novel targeted therapeutic approaches to arrest disease progression in this and other muscular dystrophies that are currently without effective treatment options.