PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutation
Shao Wei Hu, Sohyang Jeong, Luoying Jiang, Hansol Koo, Zijing Wang, Won Hoon Choi, Biyun Zhu, Heeyoung Seok, Yi Zhou, Min Gu Kim, Dan Mu, Huixia Guo, Ziyi Zhou, Sung Ho Jung, Yingting Zhang, , Yilai Shu
This study uses base editing to correct a mutation causing hearing loss in a humanized mouse model, restoring hearing function and gene expression.
bioRxiv · Oct 29, 2024 · PreprintGenetics