换一批外显子跳跃治疗Duchenne型肌营养不良症的研究进展
Advances of treatment for Duchenne muscular dystrophy with exon skipping
摘要Duchenne型肌营养不良症是一种致死性肌肉疾病,抗肌萎缩蛋白基因缺陷是导致本病的原因,目前本病尚无特效的疗法.反义寡核苷酸(antisense oligonucleotides,AOs)诱导的外显子跳跃作为一种新的治疗手段具有良好的应用前景.本文主要从外显子跳跃治疗的原理、基础研究及临床研究进行综述.
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abstractsDuchenne muscular dystrophy (DMD) is a lethal muscular disorder caused by mutations in the dystrophin gene for which no mutation-targeted therapy has been available so far. However, a new method named exon-skipping mediated by antisense oligonucleotides has considerable potential for DMD therapy. In this review, the principle, basic research and clinical research of exon-skipping are mainly summarized.
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栏目名称
DOI
10.3760/cma.j.issn.1003-9406.2011.04.010
发布时间
2011-11-11（万方平台首次上网日期，不代表论文的发表时间）
基金项目
"十一五"国家支撑计划（(2006BAI05A07)）
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