FBP21 Inhibitors

FBP21 inhibitors are a class of chemical compounds that target the splicing factor FBP21 (Formin-binding protein 21), a protein that plays a role in RNA splicing, specifically in the recognition and assembly of the spliceosome complex. FBP21 is involved in mediating interactions between various components of the spliceosome, thereby facilitating the precise removal of introns from pre-mRNA during splicing. Inhibition of FBP21 interferes with its ability to bind to other splicing factors, disrupting the proper assembly and function of the spliceosome machinery. This disruption can lead to aberrant splicing of pre-mRNA, which may result in altered gene expression and changes in the abundance of specific proteins.

Structurally, FBP21 inhibitors are often small molecules designed to bind to specific domains of the FBP21 protein, preventing its interaction with splicing-related partners. These compounds may achieve inhibition by targeting the WW domain of FBP21, a key site involved in protein-protein interactions. This domain plays a crucial role in recognizing proline-rich motifs in other proteins. By occupying this domain or otherwise altering its structure, FBP21 inhibitors effectively block the functional role of the protein in splicing. The development and characterization of these inhibitors provide valuable tools for studying the molecular mechanisms of RNA splicing and for understanding the broader role of FBP21 in cellular processes.