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Updated: Jan 28, 2026

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ALSとFTDの精密医療のためのde novo暗号スプライシングの作成

Oscar G Wilkins1,2, Max Z Y J Chien1,2, Josette J Wlaschin3,4

  • 1UCL Queen Square Motor Neuron Disease Centre, Department of Neuromuscular Diseases, UCL Queen Square Institute of Neurology, University College London, London WC1N 3BG, UK.

Science (New York, N.Y.)
|October 3, 2024
PubMed
まとめ

研究者はTDP-REGという 新しいシステムを開発し ALSのような 神経退行性疾患における タンパク質発現を制御するために 疾患特有のスプライシングを使用しています この技術は,TDP-43の機能喪失に反応することで,標的型治療のための新しい道を提供します.

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