Sydney Lu, MD, PhD

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.