Targeted therapy improves cellular dysfunction, ataxia, and seizure susceptibility in a model of a progressive myoclonus epilepsy

Huijie Feng, Jerome Clatot, Keisuke Kaneko, Marco Flores-Mendez, Eric R. Wengert, Carly Koutcher, Emily Hoddeson, Emily Lopez, Demetrius Lee, Leroy Arias, Qiansheng Liang, Xiaohong Zhang, Ala Somarowthu, Manuel Covarrubias, Martin J. Gunthorpe, Charles H. Large, Naiara Akizu, Ethan M. Goldberg