NeurosciencebioRxivPreprint
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…·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.
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