Christopher R. Vakoc, MD, PhD

Co-funded by the Dick Vitale Pediatric Cancer Research Fund and the Jeff Gordon Children’s Foundation

What big question(s) will your work answer? Rhabdomyosarcoma is a deadly cancer that occurs in children and young adults. Several decades of research points to a specific molecule (called PAX3-FOXO1) as the most compelling drug target in this disease. However, we simply do not understand the molecular details of PAX3-FOXO1 enough to made a medicine that exploits this target. The big question addressed in this project will be to understand this compelling target with atomic detail by applying innovative technology. • Why does this question matter? Children continue to die of rhabdomyosarcoma and yet the medicines used in the clinic are woefully inadequate and toxic. A new therapy tailor-made for this disease could change everything. • How will your work answer the big question? Our work has the potential to provide a basic science foundation upon which a drug discovery campaign could be launched.

Location: Cold Spring Harbor Laboratory - Cold Spring Harbor, NY
Proposal: High-resolution functional mapping and in vitro reconstitution of the PAX3-FOXO1 fusion oncoprotein complex
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