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Updated: Jul 16, 2025

Measuring RAN Peptide Toxicity in C. elegans
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一个循环罗-意米达聚胺减少了CAG/CTG三重重复神经疾病模型中的致病性RNA

Susumu Ikenoshita1,2, Kazuya Matsuo1, Yasushi Yabuki1,3

  • 1Department of Genomic Neurology, Institute of Molecular Embryology and Genetics (IMEG).

The Journal of clinical investigation
|September 14, 2023
PubMed
概括

一种新的化合物,CWG-cPIP,有效地向扩展的CAG/CTG (CWG) 重复DNA,这是神经系统疾病的关键因素,如1型肌性衰竭和亨廷顿病. 这种治疗减少了有毒的RNA和蛋白质水平,为这些疾病提供了潜在的治疗方法.

关键词:
遗传性疾病是一种遗传性疾病.神经科学是一个神经科学.没有编码的RNAs.治疗方法 治疗方法转录 转录 转录 转录

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