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Updated: Jan 16, 2026

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
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对杜申尼肌肉发育不良症的表子跳转疗法的发展和未来前景

Yasuhiro Takeshima1, Tomoko Lee1, Hideki Shimomura1

  • 1Department of Pediatrics, Hyogo Medical University School of Medicine, Japan.

Brain & development
|October 1, 2025
PubMed
概括

使用反感性寡核化物 (AS-oligo) 的外显跳转疗法通过恢复素蛋白来治疗杜申肌肉发育不良 (DMD) 的前景有望. 然而,长期疗效,早期诊断和组合疗法需要进一步研究DMD治疗.

关键词:
杜申尼肌肉发育不良症是什么迪斯特罗芬 (Dystrophin) 是一种消极蛋白质.在Exon跳过的情况下.拼接切换疗法是可以进行的.拼接 拼接 拼接 拼接

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