摘要
Duchenne型肌营养不良症(Duchenne muscular dystrophy,DMD)表现为进行性肌肉萎缩,是一种致死性、遗传性神经肌肉疾病。尽管对肌肉萎缩的分子机理的研究取得了很大进展,但是仍然不能治愈,细胞治疗是很有前景的一种治疗方法。成肌细胞移植面临的主要限制是注射后细胞分布差,免疫排斥和细胞存活率低;骨髓干细胞移植需要解决横向分化的低效率问题;而肌源性干细胞看来能更有效地再生表达dystrophin的肌纤维。
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 pmmiseful option. This review will focus on the present status of cell-based therapy. Myoblast transfer therapy is hindered by minimal distribution of ceils 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.
出处
《中华医学遗传学杂志》
CAS
CSCD
北大核心
2006年第6期659-661,共3页
Chinese Journal of Medical Genetics
