RNA base editing that avoids CRISPR helps restore hearing in mice with a mutation causing early stop signals in the Otof gene

A CRISPR-free RNA base editor restored otoferlin expression and improved hearing in mice with a specific Otof nonsense mutation.

Preclinical mouse gene-editing study delivered the RESTART v3 RNA base editor into cochleae of Otof c.1315 C > T deaf mice and measured otoferlin, hearing, and auditory startle responses.

The result is limited to a mouse model of one Otof nonsense mutation and does not yet show human safety or efficacy.