Article

Artificial Intelligence-Driven Design of Antisense Oligonucleotides for Precision Medicine in Neuromuscular Disorders

2025-09-18

Abstract excerpt

Rare neuromuscular disorders impose a significant burden on patients, caregivers, and the health care system. However, effective disease-modifying therapies remain limited. Antisense oligonucleotides (ASOs) have emerged as a promising therapeutic strategy, en-abling targeted modulation of gene expression through mechanisms such as exon skip-ping, exon inclusion, and transcript degradation. Despite the clinical app...

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Literature Corpus work: 347022ef-b52b-5c68-af11-76df8fda01a6
DOI: 10.20944/preprints202509.1588.v1

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