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…·Yulia Grishchuk
This study shows that AAV-mediated WDR45 gene transfer rescues neurologic deficits and autophagy defects in a mouse model of BPAN.