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Updated: Sep 10, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

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抑制性tRNA作为无意义突变相关病理的个性化治疗

Zoya Ignatova1, Suki Albers1

  • 1Institute of Biochemistry and Molecular Biology, University of Hamburg, Hamburg, Germany.

Pharmacology & therapeutics
|August 24, 2025
PubMed
概括

工程转移RNAs (tRNAs) 可以抑制无意义突变,这些突变会通过停止蛋白质的产生导致遗传疾病. 本综述探讨了为个性化基因治疗设计这些抑制性tRNA (超级tRNA) 的进展.

关键词:
篮子治疗方法基于RNA的疗法罕见疾病抑制器tRNAtRNA工程tRNA治疗方法

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