Precise and efficient DNA base editing restores normal hearing in adult DFNB9 mouse model - PubMed
5 hours ago
- #DFNB9
- #DNA base editing
- #hearing restoration
- Precise DNA base editing using Nme2ABE8e restored normal hearing in adult DFNB9 mouse models.
- The study identified Nme2ABE8e as the most efficient base editor for correcting the Otof c.2815C>T mutation.
- Treatment was effective in mice aged 1-4 months, indicating a broad therapeutic time window.
- Hearing restoration was sustained for at least 6 months post-treatment with no detected ototoxicity or neurological deficits.
- The findings suggest potential for a long therapeutic time window in DFNB9 patients undergoing gene therapy.