中华医学杂志（英文版）2006，Vol.119Issue(16)：1381-1391,11.
Current understanding of dystrophin-related muscular dystrophy and therapeutic challenges ahead
Current understanding of dystrophin-related muscular dystrophy and therapeutic challenges ahead
ZHOU Guang-qian 1XIE Hui-qi 2ZHANG Su-zhen 2YANG Zhi-ming2
作者信息
- 1. Centre for Cancer Research and Cell Biology, Queen's University of Belfast, Belfast, BT12 6B J, UK
- 2. Department of Stem Cell and Tissue Engineering, State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu 610041, China
- 折叠
摘要
Abstract
Objective To review the recent research progress in dystrophin-related muscular dystrophy includes X-linked hereditary Duchenne and Becker muscular dystrophies (DMD and BMD).Data sources Information included in this article was identified by searches of PUBMED and other online resources using the key terms DMD, dystrophin, mutations, animal models, pathophysiology, gene expression, stem cells, gene therapy, cell therapy, and pharmacological.Study selection Mainly original milestone articles and timely reviews written by major pioneer investigators of the field were selected.Results The key issues related to the genetic basis and pathophysiological factors of the diseases were critically addressed. The availabilities and advantages of various animal models for the diseases were described. Major molecular and cellular therapeutic approaches were also discussed, many of which have indeed exhibited some success in pre-clinical studies but at the same time encountered a number of technical hurdles, including the efficient and systemic delivery of a functional gene and myogenic precursor/stem cells to repair genetic defects.Conclusions Further understanding of pathophysiological mechanisms at molecular levels and regenerative properites of myogenic precursor/stem cells will promote the development of multiple therapeutic strategies. The combined use of multiple strategies may represent the major challenge as well as the greatest hope for the therapy of these diseases in coming years.关键词
dystrophin/muscular dystrophy/myogenic stem cells/cell therapy/gene therapyKey words
dystrophin/muscular dystrophy/myogenic stem cells/cell therapy/gene therapy分类
医药卫生引用本文复制引用
ZHOU Guang-qian,XIE Hui-qi,ZHANG Su-zhen ,YANG Zhi-ming..Current understanding of dystrophin-related muscular dystrophy and therapeutic challenges ahead[J].中华医学杂志（英文版）,2006,119(16):1381-1391,11.
