関連する実験動画

Updated: Sep 20, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

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FUS-ALSに対するアンチセンセスオリゴヌクレオチドジャシフーセン:研究者主催の多中心型オープンケースシリーズ

Neil A Shneider1, Matthew B Harms1, Vlad A Korobeynikov1

  • 1Department of Neurology, Columbia University Irving Medical Center, New York, NY, USA.

Lancet (London, England)
|May 25, 2025
PubMed
まとめ

Jacifusenは,神経変性マーカーとFUSタンパク質のレベルを低下させることで,FUS-ALSの治療に希望を示しています. アミオトロフィック横筋硬化症に対するこの潜在的な治療法のさらなる評価は進行中です.

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Characterizing Exon Skipping Efficiency in DMD Patient Samples in Clinical Trials of Antisense Oligonucleotides

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Fluorescence-Based Detection of FEN1 Nuclease Activity and Screening of Small-Molecule Inhibitors05:46

Fluorescence-Based Detection of FEN1 Nuclease Activity and Screening of Small-Molecule Inhibitors

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