Gene therapy for hereditary deafness: Progress, challenges and translational implications from OTOF to GJB2
Early inner‐ear gene‐therapy efforts, clinical and preclinical developments in OTOF targeted therapy, and current GJB2 directed strategies, including gene replacement, cell‐specific delivery, base editing, pharmacological rescue, and potential in utero intervention are summarized.
Jia-Hui Zhao, Meng-Zhao Xun, Yu Sun
· Clinical and Translational D... · 0 citations