Allele-specific antisense oligonucleotide treatment rescues atad3-associated phenotype in zebrafish
Shlomit Ezer, Shira Yanovsky‐Dagan, Amit Granit, Matthew McDougal, Taeyoung Hwang, Israel Antman, Rotem Karni, Wan Hee Yoon, Ann Saada, Adi Inbal, Tamar Harel
This study demonstrates that allele-specific antisense oligonucleotides can rescue ATAD3A-associated developmental defects in zebrafish by reducing mutant transcripts.
bioRxiv · May 23, 2026 · PreprintGenetics