Coles Chantal A, Woodcock Ian, Pellicci Daniel G, Houweling Peter J
Murdoch Children's Research Institute (MCRI), Melbourne, VIC 3052, Australia.
Faculty of Veterinary and Agricultural Sciences, The University of Melbourne, VIC 3052, Australia.
Biomedicines. 2022 Feb 24;10(3):535. doi: 10.3390/biomedicines10030535.
The lack of dystrophin in Duchenne muscular dystrophy (DMD) results in membrane fragility resulting in contraction-induced muscle damage and subsequent inflammation. The impact of inflammation is profound, resulting in fibrosis of skeletal muscle, the diaphragm and heart, which contributes to muscle weakness, reduced quality of life and premature death. To date, the innate immune system has been the major focus in individuals with DMD, and our understanding of the adaptive immune system, specifically T cells, is limited. Targeting the immune system has been the focus of multiple clinical trials for DMD and is considered a vital step in the development of better treatments. However, we must first have a complete picture of the involvement of the immune systems in dystrophic muscle disease to better understand how inflammation influences disease progression and severity. This review focuses on the role of T cells in DMD, highlighting the importance of looking beyond skeletal muscle when considering how the loss of dystrophin impacts disease progression. Finally, we propose that targeting T cells is a potential novel therapeutic in the treatment of DMD.
杜氏肌营养不良症（DMD）中肌营养不良蛋白的缺乏导致细胞膜脆弱，进而导致收缩诱导的肌肉损伤及随后的炎症反应。炎症的影响是深远的，会导致骨骼肌、膈肌和心脏纤维化，这会导致肌肉无力、生活质量下降和过早死亡。迄今为止，先天性免疫系统一直是DMD患者的主要研究重点，而我们对适应性免疫系统，特别是T细胞的了解有限。针对免疫系统一直是DMD多项临床试验的重点，并且被认为是开发更好治疗方法的关键一步。然而，我们首先必须全面了解免疫系统在营养不良性肌肉疾病中的参与情况，以便更好地理解炎症如何影响疾病进展和严重程度。本综述重点关注T细胞在DMD中的作用，强调在考虑肌营养不良蛋白缺失如何影响疾病进展时，超越骨骼肌进行研究的重要性。最后，我们提出靶向T细胞是治疗DMD的一种潜在新疗法。
