NeurosciencebioRxivPreprint
PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F
Maryna V. Ivanchenko, Daniel M. Hathaway, Eric M. Mulhall…·David P. Corey
This paper demonstrates a dual-AAV gene therapy strategy for USH1F, restoring hearing, balance, and retinal function in mouse and human models.