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

DOI Web Site Web Site 2 Citations
  • Wei-Hsi Yeh
    Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Olga Shubina-Oleinik
    Department of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
  • Jonathan M. Levy
    Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Bifeng Pan
    Department of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
  • Gregory A. Newby
    Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Michael Wornow
    Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Rachel Burt
    Murdoch Children’s Research Institute, The Royal Children’s Hospital, Parkville, VIC 3052, Australia.
  • Jonathan C. Chen
    Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.
  • Jeffrey R. Holt
    Department of Otolaryngology, F.M. Kirby Neurobiology Center, Boston Children's Hospital and Harvard Medical School, Boston, MA 02115, USA.
  • David R. Liu
    Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA 02142, USA.

Description

<jats:p>Base editing in postnatal mice repairs a recessive hearing loss mutation, rescues sensory transduction, and transiently restores auditory function.</jats:p>

Journal

  • Science Translational Medicine

    Science Translational Medicine 12 (546), eaay9101-, 2020-06-03

    American Association for the Advancement of Science (AAAS)

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