Small molecule v-ATPase inhibitor Etidronate lowers levels of ALS protein ataxin-2
Garam Kım, Lisa Nakayama, Jacob A. Blum, Tetsuya Akiyama, Steven Boeynaems, Meena Chakraborty, Julien Couthouis, Eduardo Tassoni-Tsuchida, Caitlin M. Rodriguez, Michael Cory Bassik, Aaron D. Gitler
The paper identifies Etidronate, an FDA-approved drug, as a method to lower ataxin-2 levels in neurons using a CRISPR screen and validates its efficacy in mice.
bioRxiv · Dec 20, 2021 · PreprintNeuroscience