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Updated: Sep 18, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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异构体介导的线粒体输送反意义的寡核酸

Dora von Trentini1, Ivan J Dmochowski1

  • 1Department of Chemistry, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

Nucleic acid therapeutics
|June 25, 2025
PubMed
概括

我们开发了一种使用外体来向线粒体输送2'-O-甲基RNA寡核酸的方法,用于反感性疗法. 这种方法可以通过降低有害蛋白质水平来向线粒体DNA (mtDNA) 疾病.

关键词:
2′-OMe RNA 是一种具有2′-OMe 的RNA.反感的寡核化物.基于外基因组的传递细胞外囊泡中的细胞外囊泡.胎儿牛血清 胎儿牛血清线粒体局部化的定位.

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