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, Zdeňka Ellederová, Hannah Auch, Andrea Bähr, Petra Vochozková, Janet Fadl, Vanessa Arnold, Taras Ardan, Miroslav Veith, Gianluca Santamaria, Georg Dhom, Wolfgang Hitzl, Barbara Keßler, , 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.
EMBO Molecular Medicine · Mar 7, 2022Peer-reviewedGenetics