ERD03 is a potent disruptor of the EXOSC3-RNA interaction, with a Kd of 17±7 μM . ERD03 induces PCH1B-like phenotype in zebrafish embryo and can be used for neurological disorder disease research[1].
ERD03 inhibits the binding of G-rich RNA and EXOSC3 with ~18% inhibition at 50 μM[1].
ERD03 (50 μM; 5 days) causes significant spinal curvature and recapitulates PCH1B phenotype in development in zebrafish embryos[1].
ERD03 (50 μM) induces a ~6-fold upregulation of ataxin1b mRNA and results in a minor accumulation of ataxin1a mRNA in embryos. ERD03 induces an atrop of the zebrafish cerebellum and results in zebrafish cerebella half the size of control brains in DMSO[1].
[1]. Liberty FranÇois-Moutal, et al. A Chemical Biology Approach to Model Pontocerebellar poplasia Type 1B (PCH1B). ACS Chem Biol. 2018 Oct 19;13(10):3000-3010.