RARE DISEASE RESEARCH & TRAINING
Development of CRISPR/Cas Adenosine Base Editors for Reversing a Tumor Suppressor Gene Mutation in a Rare Pediatric Brain Tumor
IPRD funded research by Qing-Xiang (Amy) Sang, Ph.D., & Boris Kantor, Ph.D.
Atypical Teratoid Rhabdoid Tumor (ATRT) is a fast‑growing brain cancer that affects very young children and has few effective treatments. This project studies a protein called SMARCA4, which helps cancer cells survive when another key gene (SMARCB1) is missing. By turning off SMARCA4 in lab‑grown tumor cells, the team will see how the cancer responds and whether it becomes more vulnerable to existing drugs. The work will also test whether blocking related proteins or combining treatments makes the cancer easier to kill. The goal is to uncover weak points in ATRT cells and lay the groundwork for safer, more targeted therapies for children.