Funded by the Dick Vitale Gala in memory of John Saunders
My research team is developing and applying novel tools for genetics, genomics and systems biology to tackle fundamental problems in cancer biology and therapeutics. In this project, we will focus on childhood cancer, the leading disease-related cause of death among children in the United States. Better treatments for these types of cancer will thus deeply benefit children. Developing such treatments will require addressing the highly complex and heterogeneous nature of cancer, for any given tumor can contain an enormous repertoire of genetic mutations within it that can also change over time. Understanding the roles of cancer genes has been hampered by the lack of large-scale methods to directly identify and interrogate the function of large numbers of genes in vivo. My goal is to invent such a platform, which will allow simultaneous mapping of the effects of many genes on cancer progression and therapeutic responses. The platform will combine genome editing, synthetic biology, in vivo animal models, high-throughput genetic screening and high-performance computing. I will develop this platform and apply it to study both solid tumors and liquid cancers diagnosed in children. I will first use it to perform large-scale in vivo genetic analysis in medulloblastoma to reveal the global genetic landscape of the progression of this disease. This analysis will also discover important driver genes, providing knowledge for more precise diagnostics and prognoses, and potentially better therapeutic strategies. Discovery of such targets will ultimately lead to improved therapeutics to save many children’s lives from childhood cancer.