School of Life Sciences, Arizona State University, Tempe, AZ 85287-4501, USA.
Molecular and Cellular Biology Graduate Program, School of Life Sciences, Tempe, AZ 85287 4501, USA.
Biomolecules. 2023 Oct 17;13(10):1536. doi: 10.3390/biom13101536.
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.
肌肉萎缩症是一组异质性的遗传性肌肉消耗疾病，根据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。肌肉萎缩症病理生理学的一个共同特征是与肌肉纤维化瘢痕形成相关的慢性炎症。随着这些疾病的进展，许多患者患有心肌病，伴有心脏组织纤维化。抗炎糖皮质激素是全球最常见的肌肉萎缩症——杜氏肌营养不良症的标准治疗方法；然而，长期暴露于糖皮质激素会导致严重的不良反应，限制了其使用。因此，开发新的药物治疗方法来限制炎症和纤维化，以减少肌肉损伤并促进修复，这一点非常重要。在这里，我们研究了肌肉萎缩症的病理生理学、遗传背景和新兴治疗策略。
