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Updated: Jul 11, 2025

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
07:44

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

Published on: September 14, 2019

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在通过CRISPR/Cas9恢复读取时,DYSF基因中出现了意想不到的额外外外突变

Olga Levchenko1, Irina Panchuk1, Konstantin Kochergin-Nikitsky1

  • 1Research Centre for Medical Genetics, 115522, Moscow, Russia.

Bio Systems
|November 9, 2023
PubMed
概括

克里斯普尔/卡斯基因编辑成功地跳过了DYSF基因中的特定外因子,有可能治疗肌肉病. 意想不到的是,跳过26-27号外子也导致了30号外子在转录中缺席,突出了更广泛的基因调节效应.

关键词:
这就是CRISPR/Cas9的作用.在 DYSF 里面,你会看到 DYSF.迪斯弗林林是什么意思?在Exon跳过的情况下,Exon跳过.基因组编辑 基因组编辑

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