Cyclopamine is a teratogenic Smo inhibitor famous for inducing cyclopia in developing mammalian fetuses. Cyclopia arises from improper differentiation in the fetus, a process heavily regulated by the Hedgehog (Hh) signaling pathway. The proteins Smo and patched mediate cellular response to the Hedgehog protein signal, and aberrant behavior of these proteins can lead to oncogenic states. While cyclopia and fetal failure are the notorious effects of cyclopamine on development, the compound has also been used to suppress and reverse the effects of oncogenic mutations in Smo and patched.
1. Taipale, J., et al., 2000. Effects of oncogenic mutations in Smoothened and Patched can be reversed by cyclopamine. Nature. 406(6799): 1005-9. PMID: 10984056
2. Berman, David M., et al., 2002. Medulloblastoma growth inhibition by hedgehog pathway blockade. Science (New York, N.Y.). 297(5586): 1559-61. PMID: 12202832
3. Chen, James K., et al., 2002. Small molecule modulation of Smoothened activity. Proceedings of the National Academy of Sciences of the United States of America. 99(22): 14071-6. PMID: 12391318
See how others have used Cyclopamine. Click on the entry to view the PubMed entry .
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