CRISPR-free RNA base editing mediated PTC-readthrough restores hearing in mice with Otof nonsense mutation

已完结 20 由 ZZY211 发布于 2026/4/7 21:55:11

DOI:10.1038/s41467-025-67112-w
作者：Hanxiao Sun, Qi Teng, Wenqing Liu, Rui Guo, Menghua Li, Wei Xiong, Qiang Huang, Qianru Yu, Nan Luo, Yang Li, Jinghui Song, Shusheng Gong, Xi Shi, Chengqi Yi, Ke Liu
文献类型：期刊论文
补充材料：仅需补充材料
文献链接：https://doi.org/10.1038/s41467-025-67112-w

Nature

Abstract：The gene therapy achieved by AAV-mediated otoferlin-overexpression is an effective therapeutic strategy for congenital deafness. However, achieving its physiological and endogenous patterns

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