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

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Author notes

1. These authors contributed equally: Hanxiao Sun, Qi Teng, Wenqing Liu, Rui Guo, Menghua Li.

Authors and Affiliations

1. The National Key Laboratory of Gene Function Studies and Manipulation, School of Life Sciences, Peking University, Beijing, China

   Hanxiao Sun, Qiang Huang, Nan Luo, Jinghui Song & Chengqi Yi

2. Department of Otolaryngology Head and Neck Surgery, Beijing Friendship Hospital, Capital Medical University, Beijing, China

   Qi Teng, Rui Guo, Menghua Li, Wei Xiong, Qianru Yu, Yang Li, Shusheng Gong & Ke Liu

3. School of Life Sciences, Tsinghua University, Beijing, China

   Wenqing Liu

4. Tsinghua-Peking Joint Center for Life Sciences, Tsinghua University, Beijing, China

   Wenqing Liu

5. Beijing Clinical Research Institute, Beijing, China

   Yang Li & Ke Liu

6. Clinical Center for Hearing Loss, Capital Medical University, Beijing, China

   Shusheng Gong & Ke Liu

7. Academician Workstation of Hainan University (Sanya), School of Pharmaceutical Sciences, Hainan University, Haikou, Hainan, China

   Xi Shi

8. Peking-Tsinghua Center for Life Sciences, Peking University, Beijing, China

   Chengqi Yi

9. Department of Chemical Biology and Synthetic and Functional Biomolecules Center, College of Chemistry and Molecular Engineering, Peking University, Beijing, China

   Chengqi Yi

10. Beijing Advanced Center of RNA Biology (BEACON), Peking University, Beijing, China

    Chengqi Yi

Contributions

K.L., C.Y., and X.S. proposed the conception of the project. H.S., W.L., Q.H., N.L., J.S., and C.Y. designed the RNA editing tools. Q.T., R.G., M.L., W.X., Q.Y., Y.L., S.G., X.S., and K.L. designed and conducted animal testing. H.S., Q.T., W.L., R.G. and M.L. collected the data and performed data analysis. H.S., Q.T., W.L., R.G. and M.L. developed photographs and schematic diagrams in the manuscript. H.S., Q.T., M.L., X.S., C.Y., and K.L. wrote the manuscript with contributions from all authors.

Corresponding authors

Correspondence to Xi Shi, Chengqi Yi or Ke Liu.

Competing interests

A patent application has been filed by Peking University for the RESTART technology disclosed in this publication; C.Y. is the inventor on the patent application. The Otof c.1315 C > T (p.R439*) mice used in this article have obtained a Chinese patent (ZL202110894148.4); K.L. and X.S. are the inventors on this patent application. The other authors declare that they have no competing interests.

Peer review information

Nature Communications thanks the anonymous reviewers for their contribution to the peer review of this work. A peer review file is available.

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