Article

Enhanced splicing modulation by NMA-modified antisense oligonucleotides

2025-10-07

Abstract excerpt

Aberrant RNA splicing contributes to many human diseases, and splice‑switching antisense oligonucleotides (SSOs) are ideally suited as a therapeutic strategy to modulate splicing and restore normal gene expression. Nusinersen (Spinraza™) has revolutionized the treatment of spinal muscular atrophy. It is a splice-switching oligonucleotide (SSO) that is modified with 2′- O -methoxyethyl (MOE) modifications. Here, w...

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Literature Corpus work: 0bba2a8f-c0e9-596b-8776-6d480664fe8f
DOI: 10.1101/2025.10.06.680653

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