UNC School of Medicine

2015-2017

RNA signatures, functions and therapeutic targets

Principal Investigator: Dr. Praveen Sethupathy, Ph.D.- Assistant Professor of Genetics

Our research is focused on: (1) validating UNC’s RNA signature of FLC in an independent set of FLC, HCC, and CCA samples (collaboration with Dr. Michael Torbenson); (2) evaluating the expression and function of these RNAs in the first ever FLC transplantable tumor line- TU-2010 (collaboration with Dr. Lola Reid); and (3) identifying candidate therapeutic targets of FLC for future clinical development. For example, effective small molecule inhibitors are already available for some of the proteins of interest, such as CA12, and these could be tested immediately if our findings suggest that CA12 is a critical driver of FLC progression. Additionally, it may be possible to identify early diagnostic markers, which is critical for survival outcome. CA12 was previously detected in the sera of patients with specific lung tumors and microRNA-10b was reported as a candidate biomarker of breast cancer. UNC hypothesizes that CA12 and microRNA-10b are present at high levels in the sera of FLC patients as well. In order to test this hypothesis, UNC requires access to FLC sera. If findings in the serum samples validate the hypothesis, these genes could serve as early diagnostic biomarkers for FLC. The proposed research is groundbreaking because it is focused on defining the molecular landscape of FLCs and identifying biomarkers and pathogenic drivers of FLCs. The successful completion of these aims will chart a clear path for subsequent efforts to develop novel early diagnostic tools and therapeutic strategies for FLCs.