Article
Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation
2023-10-28
Abstract excerpt
Mutations in microRNA-96 ( MIR96 ) cause dominant delayed onset hearing loss DFNA50 without treatment. Genome editing has shown efficacy in hearing recovery by intervention in neonatal mice, yet editing in the adult inner ear is necessary for clinical applications. Here, we developed an editing therapy for a C>A point mutation in the seed region of the Mir96 gene, Mir96 14C>A associated with hearing loss by s...
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Identifiers and source
- Literature Corpus work
- d700eae9-0332-5bab-b19c-7df3a5d34d80
- DOI
- 10.1101/2023.10.26.564008
