This project aims to provide a fast, semi-high throughput and cheap animal model for identifying and/or characterizing promising drug targets for treating desmoid tumors. We have obtained proof of principle that, in an approach of mosaic multiplexed gene knockout using CRISPR/Cas9, we can identify genes that are critical for desmoid tumor formation. Using a streamlined experimental pipeline, we will be able to rapidly assess whether a list of other genes that are highly expressed in human desmoid tumors play an essential role in tumor proliferation and could serve as anchor points for novel therapeutic drug development. In addition we will further evaluate and optimize the compound BC-2059 (BetaCat Pharma) on desmoid tumor formation in our experimental Xenopus model. The treatment protocol established with BC-2059 will serve as a blueprint for future assessment of additional compounds with the goal of testing toxicity, uptake and efficacy. We believe that our model offers a unique experimental platform that can be easily plugged into the research lines of several groups active in the field.
LAY VERSION OF ABSTRACT- “Identifying targets for therapy in a novel genetic Xenopus model for desmoid tumor formation.”