SON DNA-binding protein (SON)

SON DNA-binding protein is a large, multifunctional protein primarily localized in nuclear speckles, where it plays a critical role in pre-mRNA splicing and transcriptional regulation (UniProt P18583). It is particularly essential for the efficient splicing of weak splice sites in genes required for cell cycle progression and DNA replication (Ahn et al., 2011). Mutations in the SON gene are the primary cause of Zhu-Tokita-Takenouchi-Kim (ZTTK) syndrome, a multisystem developmental disorder characterized by intellectual disability, malformations, and seizures (Tokita et al., 2016). In oncology, SON is often overexpressed and contributes to the maintenance of cancer cell proliferation and survival, particularly in leukemia and breast cancer (Kim et al., 2016). While no clinical drugs currently target SON directly, its central role in RNA processing and its association with various malignancies highlight its importance as a research focus for novel drug development.

Other names

BASSNREBPSON3DBP-5Negative regulatory element-binding protein

Currently, there are no FDA-approved drugs specifically targeting SON. Research focuses on modulating its role in RNA splicing or its interaction with transcription machinery using antisense oligonucleotides or small molecules (Ahn et al., 2011).