ML099 is a Ras-related GTPase activator. Mutated forms of small GTPases induce proliferation and transformation of a number of cell types as well as differentiation of neuronal cells. The only known activation mechanism of small GTPase is via guanine nucleotide-exchange factors (GEFs). Data suggests that some diseases, including immunodeficienty syndromes result from loss of function mutations in the Rac2 GTPase, and the GEF cdc42. ML099 can activate cdc42, Rac1 and Ras GTPase activity with mid to high nM EC50s. GTPase activatiors could be useful tools for defining the mechanism of GEF activation of GTPases, further understanding of GTPase signaling mechanisms and study of diseases in which loss of function mutants are implicated.
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