Funded by the Dick Vitale Pediatric Cancer Research Fund
Our research focuses on a type of leukemia called B-cell acute lymphoblastic leukemia (B-ALL), which is most commonly found in children and adolescents. Despite advancements in treatment, a significant number of young patients do not respond well to existing therapies and face high risks of relapse. Our project specifically addresses those cases caused by changes in a gene called CRLF2, which are associated with poor outcomes. To understand and combat this challenging disease, we are using a cutting-edge technique called CRISPR/Cas9 to create detailed models of human blood cells that carry the same genetic changes seen in patients with CRLF2-related leukemia. These models allow us to study the disease in a controlled environment and understand the step-by-step development from the initial genetic changes in a human blood cell to full-blown leukemia. By examining these models at a microscopic level, using technologies that analyze individual cells, we aim to uncover new details about how these leukemias develop and find weak points where new drugs could intervene. Our goal is to identify new treatments that could target these leukemias more precisely and to explore ways to detect and perhaps prevent the disease before it fully develops. This research could lead to better survival rates and less suffering for children affected by this aggressive type of leukemia, providing hope for families facing this diagnosis. The knowledge gained could also help in understanding other similar types of childhood leukemias, broadening the impact of our work beyond B-ALL.
