In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness
Wei-Hsi Yeh, Olga Shubina-Oleinik, Jonathan M. Levy, Bifeng Pan, Gregory A. Newby, Michael Wornow, Rachel Burt, Jonathan C. Chen, Jeffrey R. Holt, David R. Liu
Abstract
in vivo restored inner hair cell sensory transduction and hair cell morphology and transiently rescued low-frequency hearing 4 weeks after injection. These findings provide a foundation for a potential one-time treatment for recessive hearing loss and support further development of base editing to correct pathogenic point mutations.
Topics & Concepts
Hair cellBiologyPoint mutationTransduction (biophysics)GeneticsInner earHearing lossMutationCell biologyGeneNeuroscienceMedicineBiochemistryAudiologyRNA regulation and diseaseCRISPR and Genetic EngineeringRNA and protein synthesis mechanisms