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

Int J Mol Sci · 2024 · PMC11312655 · PMID 39126066

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The formation of structured SFPQ-positive fibrils also increased in the Ulefnersen mimic-treated cells compared to the TMO-modified AO, although this did not reach statistical significance.

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