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Updated: May 13, 2025

Measuring RAN Peptide Toxicity in C. elegans
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Measuring RAN Peptide Toxicity in C. elegans

Published on: April 30, 2020

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五基重复蛋白向CUG重复RNA改善了1型肌性缩症小鼠模型中的RNA毒性

Takayoshi Imai1, Maiko Miyai2, Joe Nemoto3

  • 1EditForce Inc., Fukuoka 819-0395, Japan.

Science translational medicine
|April 16, 2025
PubMed
概括

科学家们设计了RNA结合蛋白来准1型肌性缩症 (DM1) 中的有毒RNA. 这种方法在细胞和小鼠模型中显示出治疗潜力,为治疗这种遗传性疾病提供了希望.

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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

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