Population-scale genome-wide prioritization of targets for allele-specific antisense oligonucleotide therapy design

Background: Allele-specific antisense oligonucleotides (ASOs) are increasingly used to selectively suppress transcripts containing pathogenic gain-of-function or dominant negative (GOF/DN) variants. However, most current approaches rely on patient-specific target selection of SNVs in cis with the disease-causing variant. This individualized approach may limit broader ASO applicability and could exacerbate inequities in access. Population-level selection of ASO-targetable SNVs is an alternative approach not previously explored at scale. Methods: For a curated set of >400 disease genes with puta

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