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Article

Allele-Selective Thiomorpholino Antisense Oligonucleotides as a Therapeutic Approach for Fused-in-Sarcoma Amyotrophic Lateral Sclerosis.

International journal of molecular sciences - 3 Aug 2024

Mejzini Rita, Caruthers Marvin H, Schafer Balazs, Kostov Ondrej, Sudheendran Kavitha, Ciba Marija, Danielsen Mathias, Wilton Steve, Akkari Patrick Anthony, Flynn Loren L

Abstract excerpt

Pathogenic variations in the fused in sarcoma (FUS) gene are associated with rare and aggressive forms of amyotrophic lateral sclerosis (ALS). As FUS-ALS is a dominant disease, a targeted, allele-selective approach to FUS knockdown is most suitable. Antisense oligonucleotides (AOs) are a promising therapeutic platform for treating such diseases. In this study, we have explored the potential for allele-selective...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 39126066
DOI: 10.3390/ijms25158495
PMCID: PMC11312655

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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