[7] Rho GTPases are central to dynamic actin cytoskeletal assembly and rearrangement that are the basis of cell-cell adhesion and migration.

This process is regulated by guanine nucleotide exchange factors (GEFs) which promote the exchange of bound GDP for free GTP, GTPase activating proteins (GAPs) which increase GTP hydrolysis activity, and GDP dissociation inhibitors which inhibit the dissociation of the nucleotide from the GTPase.

Cdc42 has also been shown to be required for both G1-S phase progression and mitosis, and it also modulates the transcription factors SRF, STAT3, and NFkB.

[13] Another study found that Cdc42 drives the process of initiating a metastatic tumor in a new tissue by promoting the expression of β1 integrin, an adhesion receptor known to be involved in metastasis.

A continually active form of the transcription factor was also capable of restoring endothelial insertion to cancer cells lacking Cdc42.

[15] In mice, systemic AZA197 treatment in vivo reduced primary tumor growth and prolonged survival.

[15] Therapy targeting Rho GTPase Cdc42 signaling pathways may be effective for treatment of patients with advanced colon cancer overexpressing Cdc42, and particularly those with KRAS-mutant disease.

Mutations in the CDC42 gene are responsible for the Takenouchi-Kosaki syndrome, an autosomal-dominant complex congenital developmental disorder associated with thrombocytopenia.