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优化基于2',4'-BNA/LNA的2',4'-BNA/LNA寡核酸,用于在体外的剪接调节

Takenori Shimo1, Satoshi Obika2

  • 1Graduate School of Pharmaceutical Sciences, Osaka University, Osaka, Japan.

Methods in molecular biology (Clifton, N.J.)
|July 28, 2025
PubMed
概括

这项研究优化了使用新型2'-O,4'-甲桥接核酸 (2',4'-BNA) /锁定核酸 (LNA) 化学的拼接切换寡核酸 (SSO). 该协议旨在改进人工核酸设计,以有效治疗遗传疾病.

关键词:
2′,4′-桥接核酸 (BNA) /锁定核酸 (LNA) 的核酸.杜申尼肌肉发育不良症是什么在Exon跳过的情况下,Exon跳过.在体外微型基因系统中.查检查 查检查 查检查拼接切换型寡核酸 (SSO) 是一种

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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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