Novel chemical inhibitor against SOD1 misfolding and aggregation protects neuron-loss and ameliorates disease symptoms in ALS mouse model

With ELISA-based screening, Woo et al. identify a new small molecule, PRG-A01, which can block the misfolding/aggregation of SOD1 or TDP-43 in a human neuroblastoma cell line. Its use in a mouse model of ALS results in improvement of muscle strength, motor neuron viability and mobility, highlighting...

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Bibliographic Details
Main Authors: Tae-Gyun Woo, Min-Ho Yoon, So-mi Kang, Soyoung Park, Jung-Hyun Cho, Young Jun Hwang, Jinsook Ahn, Hyewon Jang, Yun-Jeong Shin, Eui-Man Jung, Nam-Chul Ha, Bae-Hoon Kim, Yonghoon Kwon, Bum-Joon Park
Format: Article
Language:English
Published: Nature Portfolio 2021-12-01
Series:Communications Biology
Online Access:https://doi.org/10.1038/s42003-021-02862-z