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Updated: Jun 18, 2025

A Reporter Based Cellular Assay for Monitoring Splicing Efficiency
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Published on: September 15, 2021

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与深层内基序列的U1 snRNA相互作用调节脊柱肌肉缩基因多个前体的拼接

Eric W Ottesen1, Natalia N Singh1, Joonbae Seo1

  • 1Department of Biomedical Sciences, Iowa State University, Ames, IA, United States.

Frontiers in neuroscience
|August 1, 2024
PubMed
概括

一种抗U1反感性寡核酸 (ASO) 通过准深层内核序列,触发脊髓肌肉缩基因中的外显子跳转. 这揭示了一个新的拼接调节机制,有可能成为新的SMA疗法.

关键词:
这就是U1 snRNA.在U1 snRNP.在mRNA前拼接.脊柱肌肉缩 (SMA) 是一种疾病.超级迷你女装是什么意思生存运动神经元 (SMN)

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