Leveraging human genetic variation to therapeutically target hundreds of genes with dominant & dispensable disease alleles
Grace D. Ramey, Quinn T. Cowan, Akshita Saxena, Bria L Macklin, Hannah L. Watry, Xiaoyue Mei, Philip H. Dierks, Luke M. Judge, Bruce R. Conklin, John A. Capra
This paper identifies therapeutic opportunities for over 500 genes with dominant and dispensable disease alleles by enabling mutation-agnostic gene therapy through allele-specific targeting.
medRxiv · Mar 27, 2026 · PreprintGenetic & Genomic Medicine