Ribonuclease recruitment using a small molecule reduced c9ALS/FTD r(G4C2) repeat expansion in vitro and in vivo ALS models.
Jessica A BushHaruo AikawaRita FuerstYue LiAndrei UrsuSamantha M MeyerRaphael I BenhamouJonathan L ChenTanya KhanSarah Wagner-GriffinMontina J Van MeterYuquan TongHailey OlafsonKendra K McKeeJessica L Childs-DisneyTania F GendronYong-Jie ZhangAlyssa N CoyneEric T WangIlyas YildirimKye Won WangLeonard PetrucelliJeffrey D RothsteinMatthew D DisneyPublished in: Science translational medicine (2021)
[Figure: see text].