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Updated: Sep 14, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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在NMD途径激活后,通过使用拼接切换的寡核酸激活基因表达的抑制

Pierre Hirt1, Anne Marchalot2,3, Catherine Horiot2

  • 1Laboratoire Contrôle de la Réponse Immune B et des Lymphoproliférations (CRIBL), CNRS Unité Mixte de Recherche 7276, INSERM Unité 1262, Université de Limoges, Limoges, France.

Methods in molecular biology (Clifton, N.J.)
|July 23, 2025
PubMed
概括

这项研究引入了拼接切换的寡核酸 (SSO) 用于通过诱导外子跳转和转录降解来静止基因. 这种新的方法有效地降低了淋巴细胞中的增强酶同质素2 (EZH2) 表达.

关键词:
反感性寡核化物 (ASO)增强器的Zeste同类 2 (EZH2) 的增强器.在Exon跳过的情况下,Exon跳过.基因沉默是一种基因沉默.敲击击退役的情况是这样的.没有意义的mRNA衰变 (NMD)拼接切换型寡核酸 (SSO) 是一种

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