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Oral-facial-digital syndrome 1 (OFD1)

In vitro studies suggest inhibiting OFD1 could be useful for treating ciliopathies and cancers associated with aberrant ciliogenesis. Cilia are required for the proper function of disease-relevant cell signaling pathways. In mouse embryonic fibroblasts with defective ciliogenesis, ciliogenesis was restored by partial knockdown of Ofd1. In a human breast cancer cell line lacking cilia, partial knockdown of Ofd1 restored ciliogenesis in about 20% of cells. Next steps include screening for inhibitors of OFD1 and assessing whether the cilia formed on cancer cells following OFD1 depletion are functional.

SciBX 6(41); doi:10.1038/scibx.2013.1158
Published online Oct. 24, 2013

Unpatented; licensing status not applicable

Tang, Z. et al. Nature; published online Oct. 2, 2013;
Contact: Qing Zhong, The University of Texas Southwestern Medical Center, Dallas, Texas