ArticleMolecular therapy. Nucleic acids2026
Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to
Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Over the last decades, antisense oligonucleotides (AONs) have emerged as powerful tools to modulate alternative splicing, allowing exclusion of a specific exon from a target mRNA to bypass mutations or restore the reading frame, enabling production of a functional protein. In Duchenne muscular dystrophy (DMD), a lethal X-linked disorder affecting 1:5,000 males, four AON therapies have received accelerated approval. However, these treatments are applicable to only 27% of patients, leaving most individuals with rare mutations without therapeutic options. Here, we outline a methodology to identify relevant AONs for such populations. This approach aims to prequalify candidates and eliminate unsuitable leads before animal testing, reducing development time and costs. Following high-throughput screening, selected AONs are assessed for intravenous delivery compatibility and serum protein binding to support model selection and forecast human pharmacokinetics. As proof of concept, we applied this strategy to tricyclo-DNA AONs targeting an ultra-rare
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