成肌细胞移植：一种可能的严重儿科疾病的手术治疗方法。
Myoblast transplantation: a possible surgical treatment for a severe pediatric disease.
机构信息
Department of General Surgery and Surgical Specialties, University of Modena and Reggio Emilia Medical School, Surgical Clinic, 41100 Modena, Italy.
出版信息
Surg Today. 2010 Oct;40(10):902-8. doi: 10.1007/s00595-009-4242-z. Epub 2010 Sep 25.
Abstract
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.
摘要
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。
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本文引用的文献
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Transplantation. 2008 Jun 27;85(12):1791-801. doi: 10.1097/TP.0b013e31817726bc.
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Ischemic central necrosis in pockets of transplanted myoblasts in nonhuman primates: implications for cell-transplantation strategies.非人灵长类动物移植成肌细胞区域的缺血性中央坏死：对细胞移植策略的影响
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Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.细胞穿透肽-吗啉代偶联物改变杜氏肌营养不良症（DMD）的前体mRNA剪接，并在体内抑制鼠冠状病毒复制。
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Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries.反义寡核苷酸诱导的外显子跳跃与肌营养不良蛋白基因转录本：组合与化学方法
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Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse.吗啉代寡聚物介导的外显子跳跃可避免mdx小鼠发生营养不良性病理变化。
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