Ankyrin repeat and KH domain-containing protein 1 (ANKHD1)

Ankyrin repeat and KH domain-containing protein 1 (ANKHD1) is a large scaffold protein encoded on human chromosome 5q31.3 and ubiquitously expressed in major organs[1][3][5]. It contains two blocks of ankyrin repeat domains that facilitate protein–protein interactions and a KH (K-homology) domain implicated in RNA and single-stranded DNA binding[1][3][5]. ANKHD1 participates in multiple critical signaling pathways, including JAK/STAT, receptor tyrosine kinase, and Hippo/YAP/TAZ, influencing cell growth, S-phase cell cycle progression, migration, invasion, and EMT[1][3][5]. Overexpression of ANKHD1 in diverse cancer types correlates with high tumor proliferation, metastasis, poor patient survival, and increased tumor aggressiveness[1][3][5]. While no approved drugs target ANKHD1 directly, its role as a central regulator of oncogenic signaling makes it an attractive candidate for therapeutic intervention in cancers characterized by its overexpression. Note: "KH domain containing protein overexpressed in cancer 1" is not a standard gene/protein name; based on structure and function, the correct target is ANKHD1—not to be confused with KHDRBS1, which is a distinct KH domain-containing RNA-binding protein also implicated in cancer but with different molecular identity and function[2][4][6][7]. ANKHD1 is a molecular scaffold and RNA-binding protein, not a classical receptor, enzyme, or transporter[1][3][5]. No approved drugs or small molecules known to directly interact with or inhibit ANKHD1 as of current evidence[1][3][5].

Other names

Mask (Drosophila ortholog)Ankyrin repeat and single KH domain-containing protein 1human MASK1

No direct drugs; possible mechanisms if targeted would include inhibition of scaffold function in signaling pathways, suppression of RNA/protein interaction interfaces, or blocking interaction with specific oncogenic partners