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Updated: May 1, 2026

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
Published on: May 12, 2013
Targeting of Smoothened for therapeutic gain
Martial Ruat1, Lucile Hoch1, Hélène Faure1
1CNRS, Alfred Fessard Institute of Neurobiology, Laboratory of Neurobiology and Development, UPR-3294, Signal Transduction and Developmental Neuropharmacology Team, Bâtiment 33, 1 avenue de la Terrasse, F-91198 Gif-sur-Yvette, France.
Abstract:
The Smoothened (Smo) receptor is a key transducer of the Hedgehog (Hh) signaling pathway, which plays a critical role in tissue maintenance and repair. Recent studies have highlighted the therapeutic value of Smo antagonists for treating Hh-linked cancers. Research on Smo agonists indicates that these molecules are important not only for delineating canonical versus noncanonical Hh signaling but also for understanding the role of Smo in physiological and pathological conditions. This review provides an update on the potential therapeutic importance of Smo modulators, and unravels the increasing complexity of its pharmacology with regard to clinical implications.
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