A scalable platform for exon-skipping antisense oligonucleotide therapy development for inborn genetic diseases
Logan Newton, Bushra Haque, David Cheerie, Chung Ting Tsoi, Conor Klamann, Rebecca Sakaki, Thomas Qu, Lauren Verhaeghe, Yijing Liang, Ryan M. Marks, Evgueni A. Ivakine, Ashish R. Deshwar, Gregory Costain
The paper develops a scalable platform for designing exon-skipping ASOs to treat genetic diseases by identifying dispensable exons and validating their therapeutic potential.
medRxiv · Jul 31, 2026 · PreprintGenetic & Genomic Medicine