Genetic & Genomic MedicinemedRxivPreprint
A scalable platform for exon-skipping antisense oligonucleotide therapy development for inborn genetic diseases
Logan Newton, Bushra Haque, David Cheerie…·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.
DOI 10.64898/2026.07.29.26359175