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

Volume: 12, Issue: 546
Published: Jun 3, 2020
Abstract
Most genetic diseases arise from recessive point mutations that require correction, rather than disruption, of the pathogenic allele to benefit patients. Base editing has the potential to directly repair point mutations and provide therapeutic restoration of gene function. Mutations of transmembrane channel-like 1 gene (TMC1) can cause dominant or recessive deafness. We developed a base editing strategy to treat Baringo mice, which carry a...
Paper Details
Title
In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness
DOI
Published Date
Jun 3, 2020
Journal
Volume
12
Issue
546
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