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ADP-ribosylation factor-like protein 13B (ARL13B) is a small GTPase of the Ras superfamily, highly enriched in the primary cilium of vertebrate cells. It plays an essential role in cilia structure, assembly (ciliogenesis), and the regulation of ciliary signaling pathways critical for tissue development and organogenesis. ARL13B acts as a GEF for ARL3, facilitating the release of lipidated cargo proteins at the cilium. Additionally, it is involved in trafficking and spatial distribution of ciliary membrane proteins, interacting directly with tubulin in the ciliary axoneme. Mutations disrupting ARL13B function are causally associated with Joubert syndrome and other ciliopathies. ARL13B is not currently targeted by approved drugs, but its central role in cellular signaling and development makes it a key focus in genetic and molecular studies of ciliary diseases.
Not applicable: ARL13B is a protein target for genetic or mechanistic studies, not a current pharmacological target. Drugs targeting ARL13B (if any are developed) would likely act by modulating its GTPase activity, GEF activity for ARL3, or ciliary trafficking functions.
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