PIKFYVE inhibition mitigates disease in models of diverse forms of ALS.

doi:10.1016/j.cell.2023.01.005

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PIKFYVE inhibition mitigates disease in models of diverse forms of ALS.

Cell - 16 Feb 2023

Hung Shu-Ting, Linares Gabriel R, Chang Wen-Hsuan, Eoh Yunsun, Krishnan Gopinath, Mendonca Stacee, Hong Sarah, Shi Yingxiao, Santana Manuel, Kueth Chuol, Macklin-Isquierdo Samantha, Perry Sarah, Duhaime Sarah, Maios Claudia, Chang Jonathan, Perez Joscany, Couto Alexander, Lai Jesse, Li Yichen, Alworth Samuel V, Hendricks Eric, Wang Yaoming, Zlokovic Berislav V, Dickman Dion K, Parker J Alex, Zarnescu Daniela C, Gao Fen-Biao, Ichida Justin K

Abstract excerpt

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease that results from many diverse genetic causes. Although therapeutics specifically targeting known causal mutations may rescue individual types of ALS, these approaches cannot treat most cases since they have unknown genetic etiology. Thus, there is a pressing need for therapeutic strategies that rescue multiple forms of ALS. Here, we show...

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PMID: 36754049
DOI: 10.1016/j.cell.2023.01.005
PMCID: PMC10062012

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