Target intelligence / Profile preview

Rac family small GTPase 1 (RAC1)

Target
RAC1
Molecular classification
Small GTPase, G protein, Signaling protein
01

Overview

Rac family small GTPase 1 (RAC1) is a member of the Rho family of small GTPases, acting as an intracellular molecular switch to regulate diverse cellular processes. It cycles between an active GTP-bound state and an inactive GDP-bound state. RAC1 plays a central role in regulating actin cytoskeleton organization, cell growth, proliferation, survival, cell cycle progression, glucose uptake, and neuronal development. Dysregulation of RAC1 is linked to several conditions, including cancer and intellectual disability syndromes. Mutations, like P29S, can confer resistance to therapies such as BRAF inhibitors in melanoma. Its post-translational modifications, such as prenylation, are potential therapeutic targets.

Other names
Ras-related C3 botulinum toxin substrate 1
02

Mechanism of action

Inhibition of RAC1 activity or membrane association (prenylation)

03

Biological functions

Actin cytoskeleton organizationCell motilityCell growthCell proliferationCell survivalCell cycle progressionCell-cell adhesionEpithelial differentiationGlucose uptakeNeuronal developmentSynaptic plasticity
04

Disease associations

Intellectual developmental disorder autosomal dominant type 48Microcephaly-corpus callosum/cerebellar vermis hypoplasia-facial dysmorphism-intellectual disability syndromeCancerMelanomaTumorigenesisMetastasisMetabolic diseasesType 2 diabetes
05

Safety considerations

Aberrant cell migration/proliferation contributing to tumorigenesis/metastasisPotential for off-target effects due to ubiquitous expression
06

Interacting drugs

BRAF inhibitors

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