PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutation

PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutation

Abstract Hearing loss is one of the most prevalent sensory disorders, but no commercial biological treatments are currently available. Here, we identify an East Asia-specific founder mutation, the homozygous c.220 C > T mutation in MPZL2, that contributes to a significant proportion of hereditary...

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Main Authors: 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, Ho Byung Chae, Liheng Chen, Sung-Yeon Lee, Luo Guo, Myung-Whan Suh, Yang Xiao, Moo Kyun Park, Honghai Tang, Jae-Jin Song, Xi Chen, Ai Chen, Jun Ho Lee, Sangsu Bae, Sang-Yeon Lee, Yilai Shu
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-62562-8
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https://doi.org/10.1038/s41467-025-62562-8

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