Department of General Surgery and Surgical Specialties, University of Modena and Reggio Emilia Medical School, Surgical Clinic, 41100 Modena, Italy.
Surg Today. 2010 Oct;40(10):902-8. doi: 10.1007/s00595-009-4242-z. Epub 2010 Sep 25.
Duchenne muscular dystrophy (DMD) is a genetic X-linked recessive orphan disease that affects approximately 1 in 3 500 male births. Boys with DMD have progressive and predictable muscle destruction due to the absence of dystrophin, a protein present under the muscle fiber membrane. This absence induces contraction-related membrane damage and activation of inflammatory necrosis and fibrosis, leading to cardiac/diaphragmatic failure and death. The authors support the therapeutic role of myoblast transplantation in DMD, and describe the history and rationale for such an approach.
杜氏肌营养不良症（DMD）是一种遗传性 X 连锁隐性罕见疾病，影响约每 3500 名男婴中的 1 名。患有 DMD 的男孩由于缺乏肌营养不良蛋白，导致肌肉纤维膜下的蛋白质缺失，从而出现进行性和可预测的肌肉破坏。这种缺失会导致与收缩相关的膜损伤以及炎症性坏死和纤维化的激活，导致心脏/膈肌衰竭和死亡。作者支持肌母细胞移植在 DMD 中的治疗作用，并描述了这种方法的历史和原理。
