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Updated: Jul 5, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Published on: May 11, 2018

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通过反意义寡核酸在衰退性 Dystrophic Epidermolysis Bullosa Bullosa 中通过拼接调节

Stefan Hainzl1, Lisa Trattner1, Bernadette Liemberger1

  • 1EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology and Allergology, University Hospital of the Paracelsus Medical University, 5020 Salzburg, Austria.

International journal of molecular sciences
|January 23, 2024
PubMed
概括

反感性寡核酸 (ASO) 可以通过向 COL7A1 基因来纠正衰退性缩性表皮溶解 (RDEB) 中的错误拼接. 这种方法在恢复功能性VII型原体和改善RDEB患者皮肤完整性方面显示出希望.

关键词:
在 COL7A1 中使用.反感的寡核化物.衰退性消化性表皮溶解 牛肉拼接模块化的拼接模块化

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