Targeting Medulloblastoma by Regulating RNA Binding Proteins

The Schnepp laboratory focuses on aggressive solid tumors including high-risk neuroblastoma, rhabdomyosarcoma and medulloblastoma. Adult tumors of these types tend to have more mutations that can in turn be targeted by therapies than their pediatric counterparts. This verifies the need to identify additional druggable targets in the pediatric forms of these tumors. Previous laboratory work has identified the RNA-binding proteins (RBP) LIN28B and Musashi 2 (MSI2) as drivers of neuroblastoma. This leads researchers to believe that RBPs may be involved in other cancers as well. Prior studies have identified several RBPs as crucial to tumor development in medulloblastoma cell lines. Based on this preliminary data, researchers hypothesize that RBPs may play critical roles in medulloblastoma development. This project aims to be a comprehensive investigation of the role of RBPs, with the eventual goal of nominating targets for the treatment of these tumors and others. Access to cell lines from the Children’s Brain Tumor Network allows researchers to assess RBP vulnerabilities in as many models as possible, increasing the efficiency and impact of this project.