Allele-specific correction of ATAD3A pathogenic variants via template-free CRISPR-Cas9 editing and gene conversion
Taejeong Bae, Yohan Park, Anel LaGrone, Milovan Šuvakov, Ping Zhang, Hyekyung Park, Holly Van Remmen, James R. Lupski, Tamar Harel, Ji‐Eun Kim, Alexej S. Abyzov, Wan Hee Yoon
This study corrects a pathogenic ATAD3A variant in iPSCs using CRISPR-Cas9-induced gene conversion without donor templates, showing high efficiency and dependence on HR proteins.
bioRxiv · Oct 23, 2025 · PreprintGenomics