Article

A scalable platform for exon-skipping antisense oligonucleotide therapy development for inborn genetic diseases

2026-07-31

Abstract excerpt

<h4>ABSTRACT</h4> Antisense oligonucleotides (ASOs) are a versatile therapeutic modality for inborn genetic diseases. ASOs can induce skipping of “dispensable” exons containing disease-causing variants to rescue protein amount and function, but this approach has been studied for only a small number of genes. We developed a high-throughput in silico tool for assessing exon dispensability and designing exon-skippi...

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Literature Corpus work: 2bbb3b52-ccff-59fe-9c8e-db239bacbabd
DOI: 10.64898/2026.07.29.26359175

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