PDF(Pubmed)
Abstract：
Muscular dystrophies are a heterogeneous group of genetic muscle-wasting disorders that are subdivided based on the region of the body impacted by muscle weakness as well as the functional activity of the underlying genetic mutations. A common feature of the pathophysiology of muscular dystrophies is chronic inflammation associated with the replacement of muscle mass with fibrotic scarring. With the progression of these disorders, many patients suffer cardiomyopathies with fibrosis of the cardiac tissue. Anti-inflammatory glucocorticoids represent the standard of care for Duchenne muscular dystrophy, the most common muscular dystrophy worldwide; however, long-term exposure to glucocorticoids results in highly adverse side effects, limiting their use. Thus, it is important to develop new pharmacotherapeutic approaches to limit inflammation and fibrosis to reduce muscle damage and promote repair. Here, we examine the pathophysiology, genetic background, and emerging therapeutic strategies for muscular dystrophies.
摘要：
肌营养不良是一组异质性的遗传性肌肉萎缩疾病，根据受肌肉无力影响的身体区域以及潜在基因突变的功能活性进行细分。肌营养不良的病理生理学的共同特征是与用纤维化瘢痕替代肌肉块相关的慢性炎症。随着这些疾病的进展，许多患者患有心肌组织纤维化的心肌病。抗炎糖皮质激素代表Duchenne型肌营养不良症的护理标准，世界上最常见的肌肉萎缩症；然而，长期接触糖皮质激素会导致严重的副作用，限制其使用。因此，重要的是开发新的药物治疗方法来限制炎症和纤维化，以减少肌肉损伤和促进修复。这里,我们检查病理生理学，遗传背景,以及新兴的肌营养不良治疗策略。
