GeneticsEMBO Molecular MedicinePeer-reviewed
Early Disruption of Photoreceptor Cell Architecture and Loss of Vision in a Humanized Pig Model of Usher Syndrome
Sophia Grotz, Jessica Schäfer, Kirsten A. Wunderlich…·Nikolai Klymiuk
This study creates a humanized pig model of Usher syndrome type 1, showing disrupted photoreceptor architecture and vision loss, and validates gene therapy approaches.
49 citations