Article

Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles

2026-03-27

Abstract excerpt

Here we identify a novel therapeutic opportunity for over 500 genes with putative “dominant & dispensable” (D&D) disease alleles. In these haplo sufficient genes, a single functional allele may be sufficient for health, presenting the opportunity for therapeutic approaches that silence the pathogenic allele. We show that allele-specific targeting of common heterozygous genetic variation linked to D&D alleles enab...

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Literature Corpus work: d4965577-74cf-5e47-aa12-8e9802057e96
DOI: 10.64898/2026.03.26.26349431

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