Epigenetic Drivers in Rhabdomyosarcoma

University of Washington, Seattle, WA

Rhabdomyosarcoma (RMS) is a devastating pediatric sarcoma. There is still no effective treatment for children with relapsed or wide-spread disease. In contrast to adult cancers, RMS has fewer DNA mutations, suggesting that other molecular changes may be driving tumor growth and progression. We have identified 40 potential candidate genes that function to alter DNA structure and packaging in the cells without causing DNA mutations. Previous data suggest that these genes may play a role in RMS. Some of these genes may work together to exert biological effects. My proposed research will apply genome engineering technology to target each individual candidate gene and gene combinations in RMS cells to identify the ones that are essential for RMS tumor growth. New targets identified from the study will be the basis for novel therapy designs to improve survival of RMS patients with advanced disease.