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Edición precisa y eficiente de bases de ADN restaura la audición normal en el modelo murino adulto de DFNB9

Ziyu Zhang¹, Man Wang¹, Fangzhi Tan¹

¹Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, State Key Laboratory of Digital Medical Engineering, Jiangsu Provincial Key Laboratory of Critical Care Medicine, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Southeast University, Nanjing 210096, China.

Med (New York, N.Y.)

|February 28, 2026

Resumen

La edición genética restauró con éxito la audición en ratones deficientes en OTOF, demostrando una amplia ventana terapéutica para el tratamiento de la pérdida auditiva DFNB9. Este enfoque de editor de bases de adenina muestra resultados prometedores para futuras terapias génicas.

Palabras clave:

terapia génica AAV DFNB9 edición de bases de ADN mutación Otof cóclea AAV dual restauración de la audición pérdida auditiva hereditaria oído interno investigación preclínica ventana de tiempo terapéutica

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