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[杜氏肌营养不良症的细胞疗法]

[Cell therapy for Duchenne muscular dystrophy].

作者信息

Zhou Chang, Zhang Cheng

机构信息

Department of Neurology, Affiliated Hospital for Sun-Yat-sen University, Guangzhou, Guangdong, 510080, P. R. China.

出版信息

Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2006 Dec;23(6):659-61.

Abstract

Duchenne muscular dystrophy (DMD) is a fatal, genetic neuromuscular disorders that manifests as progressive muscle wasting. Although there has been enormous progress in the studies of the molecular mechanism of muscular dystrophy, there is still no cure. Cell-based therapy is a promiseful option. This review will focus on the present status of cell-based therapy. Myoblast transfer therapy is hindered by minimal distribution of cells after injection, immune rejection, and poor cell survival. The drawback of bone marrow-derived stem cell transplantation is the low efficiency of transdifferentiation. Compared with them, the injection of postnatal muscle-derived stem cells (MDSC) results in a superior regeneration of dystrophin-expressing myofibers.

摘要

杜氏肌营养不良症(DMD)是一种致命的遗传性神经肌肉疾病,表现为进行性肌肉萎缩。尽管在肌肉营养不良症分子机制的研究方面取得了巨大进展,但仍然没有治愈方法。基于细胞的疗法是一种有前景的选择。本综述将聚焦于基于细胞疗法的现状。成肌细胞移植疗法受到注射后细胞分布极少、免疫排斥以及细胞存活率低的阻碍。骨髓来源干细胞移植的缺点是转分化效率低。与它们相比,注射出生后肌肉来源的干细胞(MDSC)能使表达抗肌萎缩蛋白的肌纤维实现更好的再生。

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