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Updated: Jul 27, 2025

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
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Published on: May 24, 2016

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主编辑策略,以调解在DMD基因中的外显子跳转

Cedric Happi Mbakam1,2, Jeanne Roustant3, Joel Rousseau1,2

  • 1CHU de Québec Research Centre, Laval University, Québec, QC, Canada.

Frontiers in medicine
|June 12, 2023
PubMed
概括

总编辑成功地纠正了DMD基因中的移突变,恢复了杜申尼肌肉发育不良模型中的dystrophin表达. 这种基因编辑方法为这种罕见的遗传疾病提供了有前途的治疗策略.

关键词:
这就是CRISPR-Cas9的特征.在DMD基因中,DMD基因是最重要的.杜申尼肌肉发育不良症 杜申尼肌肉发育不良症突出跳过的突出跳过首席编辑主要编辑拼接地点 拼接地点

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