Stedman H H
Department of Surgery and Institute for Human Gene Therapy, University of Pennsylvania School of Medicine, Philadelphia 19104-6160, USA.
Curr Opin Mol Ther. 2001 Aug;3(4):350-6.
The muscular dystrophies are a heterogeneous group of heritable disorders in which progressive muscle degeneration leads to regional or generalized weakness. Recent advances in molecular genetics, cell biology and vector discovery have improved the outlook for therapeutic intervention. This review focuses on novel approaches to the study of disease pathogenesis and refinements in gene- and cell-based strategies for protein restoration in Duchenne and limb-girdle muscular dystrophy, and concludes with a brief discussion of priorities for future clinical investigation.
肌肉营养不良症是一组遗传性疾病，其特征为进行性肌肉退化导致局部或全身性肌无力。分子遗传学、细胞生物学和载体研究方面的最新进展改善了治疗干预的前景。本综述重点关注疾病发病机制研究的新方法，以及杜兴氏肌营养不良症和肢带型肌营养不良症中基于基因和细胞的蛋白质恢复策略的改进，并在结尾简要讨论了未来临床研究的重点。
