CRISPR-based editing strategies to rectify EYA1 complex genomic rearrangement linked to haploinsufficiency
Yi Hwalin, Yejin Yun, Won Hoon Choi, Hye‐Yeon Hwang, Ju Hyuen Cha, Heeyoung Seok, Jae‐Jin Song, Jun Ho Lee, Seung Ha Oh, Sang‐Yeon Lee, Daesik Kim
The paper develops CRISPR-based strategies to correct a complex genomic rearrangement in EYA1 linked to haploinsufficiency, showing successful gene expression restoration in patient-derived cells.
medRxiv · Nov 14, 2023 · PreprintGenetic & Genomic Medicine