Product Description: CB096 binds the 5′CGG/3′GGC internal loop structure of r(G4C2)exp RNA, interrupts its gain-of-function mechanism, and thus results in the regulation of nucleolar focusing, RNA splicing defects, RNA metabolism, nucleocytoplasmic transport dysfunction, and toxic dipeptide repeats (DPRs) generated by RNA translation. CB096 inhibits the RAN translation with an IC50 of 20 μM in HEK293T cells[1].
Applications: Neuroscience-Neuromodulation
Formula: C14H11N3O4
References: [1]Ursu A, et al., Structural Features of Small Molecules Targeting the RNA Repeat Expansion That Causes Genetically Defined ALS/FTD. ACS Chem Biol. 2020 Dec 18;15(12):3112-3123.
CAS Number: 108883-90-5
Molecular Weight: 285.25
Research Area: Neurological Disease
Target: DNA/RNA Synthesis