Research ArticleGENE EDITING

In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness

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Science Translational Medicine  03 Jun 2020:
Vol. 12, Issue 546, eaay9101
DOI: 10.1126/scitranslmed.aay9101

Article Information

vol. 12 no. 546

PubMed: 
Print ISSN: 
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History: 
  • Received for publication July 28, 2019
  • Accepted for publication April 5, 2020
  • .

Author Information

  1. Wei-Hsi Yeh1,2,3,*,
  2. Olga Shubina-Oleinik4,*,
  3. Jonathan M. Levy1,2,
  4. Bifeng Pan4,
  5. Gregory A. Newby1,2,
  6. Michael Wornow1,2,
  7. Rachel Burt5,
  8. Jonathan C. Chen1,2,
  9. Jeffrey R. Holt4,6, and
  10. David R. Liu1,2,7,
  1. 1Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  2. 2Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA.
  3. 3Program in Speech and Hearing Bioscience and Technology, Harvard Medical School, Boston, MA 02115, USA.
  4. 4Department of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
  5. 5Murdoch Children’s Research Institute, The Royal Children’s Hospital, Parkville, VIC 3052, Australia.
  6. 6Department of Neurology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
  7. 7Howard Hughes Medical Institute, Harvard University, Cambridge, MA 02138, USA.
  1. Corresponding author. Email: jeffrey.holt{at}childrens.harvard.edu (J.R.H.); drliu{at}fas.harvard.edu (D.R.L.)
  • * These authors contributed equally to this work.

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