Small molecule inhibition of PIKFYVE kinase rescues gain- and loss-of-function C9ORF72 ALS/FTD disease processes in vivo
Kim A. Staats, Celest Wen Ting Seah, Ali Sahimi, Yaoming Wang, Nicole Koutsodendris, Sung‐Jan Lin, D. Kim, W-H. Chang, Kimberly A Gray, Yuanjie Shi, Yichen Li, Morgan Chateau, Vamshidhar R. Vangoor, K. Senthilkumar, R. Jeroen Pasterkamp, , Justin K. Ichida
This study shows that inhibiting PIKFYVE kinase rescues both gain- and loss-of-function C9ORF72 disease mechanisms in mouse models of ALS/FTD.
bioRxiv · Jun 28, 2019 · PreprintNeuroscience