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Updated: Feb 20, 2026

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
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Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

Published on: May 24, 2016

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一种抗体-寡核酸结合物用于1型肌性发育不良

Nicholas E Johnson1, Li-Jung Tai2, Johanna I Hamel3

  • 1Virginia Commonwealth University, Richmond.

The New England journal of medicine
|February 18, 2026
PubMed
概括

德尔帕西巴特 (del-desiran) 在治疗1型肌性缩症方面表现有前途,通过减少有毒的mRNA和改善拼接模式. 尽管观察到一些严重的不良事件,但需要进一步进行临床研究.

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