In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness
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- Wei-Hsi Yeh
- Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
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- Olga Shubina-Oleinik
- Department of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
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- Jonathan M. Levy
- Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
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- Bifeng Pan
- Department of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
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- Gregory A. Newby
- Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
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- Michael Wornow
- Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
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- Rachel Burt
- Murdoch Children’s Research Institute, The Royal Children’s Hospital, Parkville, VIC 3052, Australia.
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- Jonathan C. Chen
- Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
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- Jeffrey R. Holt
- Department of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
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- David R. Liu
- Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
説明
<jats:p>Base editing in postnatal mice repairs a recessive hearing loss mutation, rescues sensory transduction, and transiently restores auditory function.</jats:p>
収録刊行物
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- Science Translational Medicine
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Science Translational Medicine 12 (546), eaay9101-, 2020-06-03
American Association for the Advancement of Science (AAAS)