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Updated: Sep 8, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
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アミオトロフィック横筋硬化症におけるアンチセンセスオリゴヌクレオチド療法

Gergo Erdi-Krausz1,2, Pamela J Shaw1,2

  • 1Sheffield Institute for Translational Neuroscience, the University of Sheffield.

Current opinion in neurology
|August 20, 2025
PubMed
まとめ

アンチセンセスオリゴヌクレオチド (ASO) 治療は,アミオトロフィック横筋硬化症 (ALS),特にSOD1-ALSの治療に有望である. 進行中の研究は,他の遺伝子変異と散発性ALSのためにASOを探索し,潜在的に神経変異を逆転させる.

キーワード:
アミオトロフィック・ラテラル・スクレロスアンチセンス オリゴヌクレオチド

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