AAV-mediated gene transfer of WDR45 corrects neurologic deficits in the mouse model of beta-propeller protein-associated neurodegeneration
Maria Carla Carisi, Claire E. Shamber, Martha Bishop, Madison Sangster, Uma Chandrachud, Brandon L. Meyerink, Louis‐Jan Pilaz, Yulia Grishchuk
This study shows that AAV-mediated WDR45 gene transfer rescues neurologic deficits and autophagy defects in a mouse model of BPAN.
bioRxiv · Jun 18, 2024 · PreprintNeuroscience