NeurosciencebioRxivPreprint
Small molecule v-ATPase inhibitor Etidronate lowers levels of ALS protein ataxin-2
Garam Kım, Lisa Nakayama, Jacob A. Blum…·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.