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Improved gene editing restores hearing in a mouse model of inherited deafness
Updated
Abstract
Essence
An optimized adenine base editor restored auditory function in a mouse model.
Evidence
This preclinical in vitro and mouse base-editing study screened editors for a Pou4f3 allele, achieved up to 48.5% in vitro editing, and used Anc80L65 AAV delivery in neonatal mice to recover hearing for at least four months.
Caveat
The result is limited to a neonatal mouse model with short follow-up and biosafety analyses, not clinical proof in humans.
Simplified
Key numbers
14.2 ± 2.5%
Editing Efficiency
-level editing efficiency in neonatal mouse cochleae.
60 dB SPL
Auditory Threshold
Average threshold in treated ears.
4 months
Duration of Hearing Recovery
Duration of stable hearing recovery post-treatment.
CRISPR Gene Editing