Scaling haplospecific antisense oligonucleotides from N-of-1 to broad use in genetic disease populations by diplotyping
Olivia Kim McManus, Pagé C. Goddard, Shahad Olsson, Liana Protopsaltis, Joseph G. Gleeson, Qing Zhang, Nafeesa Kahn, Ali Crawford, Stephen Francis Kingsmore
The paper identifies haplospecific antisense oligonucleotides for genetic disorder treatment using population genome sequencing and haplotyping.
medRxiv · Feb 4, 2026 · PreprintGenetic & Genomic Medicine