MICA: The role of MMP inhibitors in ameliorating muscular dystrophy

肌肉营养不良是一组遗传性疾病，表现为进行性肌肉萎缩和功能丧失，因此受影响的人会被轮椅束缚并过早死亡。虽然帮助呼吸肌和心脏的医疗既可以延长寿命，又可以提高生活质量，但没有任何治疗方法可以防止不可避免的渐进性肌肉丧失和随之而来的独立性丧失。肌肉损伤的反复循环会导致炎症和疤痕组织的形成，这很可能会抑制有效的肌肉修复。该项目将测试与这种疤痕形成过程相关的一种酶的特定抑制剂在两种肌营养不良小鼠模型中改变疾病进展的能力。酶抑制剂是由阿斯利康开发的，并在针对另一种疾病的临床试验中进行了测试，因此人们对这种药物的安全性有很多了解。因此，如果它能有效地改善小鼠的肌肉功能，它可以迅速进入肌肉营养不良的临床试验，避免通常与药物开发过程相关的拖延。

Muscular dystrophies are a set of inherited conditions that show progressive muscle wasting and loss of function such that affected individuals become wheelchair bound and suffer premature death. While medical treatment to aid the respiratory muscles and the heart both prolongs lifespan and improves quality of life, there are no treatments that prevent the inevitable progressive loss of muscle and the consequent loss of independence. The repeated cycles of muscle damage cause inflammation and scar tissue formation and this very likely inhibits effective muscle repair. This project will test the ability of a specific inhibitor of one of the enzymes associated with this scarring process to alter the disease progression in two mouse models of muscular dystrophy.The enzyme inhibitor was developed and tested in clinical trial for a different disease by AstraZeneca and so a lot is known about the safety of this drug. Therefore if it is effective in improving muscle function in the mice, it could be rapidly taken into clinical trial for muscular dystrophy avoiding the delays normally associated with the drug development process.