Sydney Lu, MD, PhD

Stanford Medical School - 2024

Novel approaches to therapeutic targets for SF3B1 mutated leukemias

Many leukemias have mutations in the SF3B1 gene. Its function is to “stitch together” RNA correctly. RNA is akin to a manufacturing blueprint for making proteins. When mutated, SF3B1 makes abnormal RNA and thus abnormal proteins. We are studying how to target these abnormal RNAs and proteins to treat leukemia.