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Dock1 shRNA is a short hairpin RNA-based therapeutic candidate designed to knock down the expression of Dedicator of cytokinesis 1 (DOCK1), a guanine nucleotide exchange factor (GEF) for Rac1 and Rac2. In the context of acute myeloid leukemia (AML) harboring NPM1 and cohesin mutations, DOCK1 is uniquely upregulated and promotes leukemic cell survival and growth through the activation of the Rac2 pathway. Preclinical studies have demonstrated that silencing DOCK1 using shRNA leads to reduced cell growth, impaired adhesion, and increased apoptosis in specific leukemic cell lines, as well as prolonged latency in transplant models. This approach highlights DOCK1 as a precision oncology target for specific genetic subsets of AML.
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