Article
A base editor for the long-term restoration of auditory function in mice with recessive profound deafness.
Nature biomedical engineering - 1 Jan 2025
Cui Chong, Wang Shengyi, Wang Daqi, Zhao Jingjing, Huang Bowei, Zhu Biyun, Chen Yuxin, Tang Honghai, Han Yu, Ye Cheng, Mu Dan, Zhang Chengdong, Yang Yuan, Bao Yihan, Lv Jun, Han Shuang, Li Geng-Lin, Li Huawei, Shu Yilai
Abstract excerpt
A prevalent recessive mutation (c.2485C>T, p.Q829X) within the OTOF gene leads to profound prelingual hearing loss. Here we show that in Otof mice harbouring a mutation (c.2482C>T, p.Q828X) homozygous to human OTOF that faithfully mimics the hearing-loss phenotype, a base editor (consisting of the deaminase ABE7.10max and the Cas9 variant SpCas9-NG) packaged in adeno-associated viruses and injected into the inner...
Read the complete abstract on PubMedTopics
- Animals
- Mice
- Humans
- Membrane Proteins
- Deafness
- Gene Editing