RNA interference (RNAi) is a widely conserved and naturally occurring process in which small RNAs lead to targeted gene silencing. In the short time since its discovery in the round worm Caenorhabditis elegans, RNAi has revolutionized our understanding of gene regulation and has been found to play key roles in viral defense mechanisms and in tumor suppression. Furthermore, researchers are actively developing RNAi-based drugs to silence disease-causing genes in patients. Some of these drugs are already in Phase II clinical trials. This new class of drugs has great potential for the treatment of childhood cancers including Ewing’s sarcoma and acute lymphoblastic leukemia, which are associated with specific cancer-causing genes that can be targeted by RNAi. The biggest challenge facing the effective use of RNAi-based therapies is safe delivery of these drugs to their proper targets while avoiding off-target effects. RNAi delivery is very efficient in C. elegans, and an important RNA transport protein discovered in the worm has been shown to be important for delivery of guide RNAs in humans. Our goal is to use C. elegans to identify and characterize additional proteins important for the delivery of RNAi. This work will allow for subsequent detailed analyses of corresponding human genes and will provide important insights for the delivery of RNAi-based therapies.
About Jennifer Whangbo, MD, PhD
Dr. Whangbo received her Bachelor's in Science from the University of North Carolina, Chapel Hill in 1994. She received her PhD from the University of California, San Francisco in 1999 in Biochemistry and her MD from University of California, Los Angeles in 2003.