Figure 1.

Pipeline for transforming insights from disease models into potential therapeutics for craniofacial disorders. Human genetic studies identify critical genes linked to craniofacial disease. Mechanistic studies, using primary patient cells, patient-specific iPS cells, and/or animal models, probe the disease gene’s role in craniofacial biology. Once the biological function of the gene is discovered, therapeutic targets can be identified. Having an in-depth view of the target’s biology aids in selecting therapeutic modalities, such as biologics, small molecules, stems cells, and possibly gene editing.