Modulating CCTG repeat expansion toxicity in DM2 Drosophila model through TDP1 inhibition

Modulating CCTG repeat expansion toxicity in DM2 Drosophila model through TDP1 inhibition

Abstract Myotonic dystrophy type 2 (DM2), caused by CCTG repeat expansion, is a common adult-onset disorder characterized by myotonia and progressive muscle degeneration with no effective treatment. Here, we identified Tyrosyl-DNA phosphodiesterase 1 (TDP1) as a novel modifier for DM2 therapeutic in...

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Main Authors: Yingbao Zhu, Shengwei Xiao, Xinxin Guan, Haitao Deng, Liqiang Ai, Kaijing Fan, Jin Xue, Guangxu Li, Xiaoxue Bi, Qiao Xiao, Yuanjiang Huang, Lin Jiang, Wen Huang, Peng Jin, Ranhui Duan
Format: Article
Language:English
Published: Springer Nature 2025-03-01
Series:EMBO Molecular Medicine
Subjects:
DM2
CCTG Repeat Expansion
Chemical Screen
TDP1
Repeat Instability
Online Access:https://doi.org/10.1038/s44321-025-00217-3
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https://doi.org/10.1038/s44321-025-00217-3

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