Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

Xue Gao, Yong Tao, Veronica Lamas, Mingqian Huang, Wei-Hsi Yeh, Bifeng Pan, Yu-Juan Hu, Johnny H. Hu, David B. Thompson, Yilai Shu, Yamin Li, Hongyang Wang, Shiming Yang, Qiaobing Xu, Daniel B. Polley, M. Charles Liberman, Wei-Jia Kong, Jeffrey R. Holt, Zheng-Yi Chen () and David R. Liu ()
Additional contact information
Xue Gao: Harvard University
Yong Tao: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
Veronica Lamas: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
Mingqian Huang: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
Wei-Hsi Yeh: Harvard University
Bifeng Pan: F.M. Kirby Neurobiology Center Boston Children’s Hospital and Harvard Medical School
Yu-Juan Hu: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
Johnny H. Hu: Harvard University
David B. Thompson: Harvard University
Yilai Shu: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
Yamin Li: Tufts University
Hongyang Wang: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
Shiming Yang: Key Lab of Hearing Impairment Science of Ministry of Education, Key Lab of Hearing Impairment Prevention and Treatment of Beijing City, Chinese PLA Medical School
Qiaobing Xu: Tufts University
Daniel B. Polley: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
M. Charles Liberman: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
Wei-Jia Kong: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Jeffrey R. Holt: F.M. Kirby Neurobiology Center Boston Children’s Hospital and Harvard Medical School
Zheng-Yi Chen: Harvard Medical School and Eaton Peabody Laboratory, Massachusetts Eye and Ear Infirmary
David R. Liu: Harvard University

Nature, 2018, vol. 553, issue 7687, 217-221

Abstract: CRISPR–Cas9 genome editing is used to correct a dominant-negative mutation in a mouse model of inherited deafness, resulting in improvements in cochlear function and hearing.

Date: 2018
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DOI: 10.1038/nature25164

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