PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F
Maryna V. Ivanchenko, Daniel M. Hathaway, Eric M. Mulhall, Kevin T. A. Booth, Mantian Wang, Cole W. Peters, Alex J. Klein, Xinlan Chen, Yaqiao Li, Bence György, 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.
bioRxiv · Nov 9, 2023 · PreprintNeuroscience