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Updated: Jul 17, 2025

Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides
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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

Published on: May 7, 2020

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抗意义寡核酸介导的DMD异子跳转效率与侧边内子保留时间和异子内的目标位置相关

Remko Goossens1, Nisha Verwey1, Yavuz Ariyurek1,2

  • 1Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.

RNA biology
|September 5, 2023
PubMed
概括

对杜氏肌肉发育不良症 (DMD) 的反感性寡核酸 (AON) 疗法显示出改善的外跳转效率. 针对5'末端附近的外显子和那些由缓慢拼接的内显子先导的外显子,可以提高DMD的治疗结果.

关键词:
在AON AON上在ASO的基础上,ASO是ASO.反意义的寡核化物.在 DMD DMD 里面,你会发现杜氏肌肉发育不良症 杜氏肌肉发育不良症迪斯特罗芬 (Dystrophin) 是一种消极蛋白质.在Exon跳过的情况下,Exon跳过.

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