Natasha J. Caplen, Ph.D.

Dr. Caplen received her Ph.D. from the University of London (Kings College Hospital Medical School), where her studies investigated the genetics of type I diabetes and its complications. Dr. Caplen's post-doctoral training began at St Mary's Hospital Medical School, Imperial College, where she focused on the development of gene therapy approaches for cystic fibrosis (CF). As a post-doctoral fellow, Dr. Caplen made significant contributions to the first pre-clinical and clinical studies of lipid-mediated gene therapy for CF. These early studies paved the way for ongoing efforts to develop gene therapy strategies for CF and other genetic and acquired diseases, including cancer. In 1996, Dr. Caplen came to the National Human Genome Research Institute (NHGRI) at NIH as a Visiting Fellow, where she initially conducted studies investigating hybrid viral vector systems for the delivery of genes. It was while at NHGRI that Dr. Caplen developed a research interest in the newly identified gene silencing mechanism, RNA interference (RNAi) leading to studies that contributed to establishing the presence of RNAi in mammalian cells. Dr. Caplen joined CCR, NCI in 2004 as a Senior Scientist, where she pioneered approaches for exploiting RNAi to investigate cancer biology and treatment and helped establish a trans-NIH facility for genome-wide RNAi screening. In January 2016, Dr. Caplen became a Tenure-Track Investigator in CCR’s Genetics Branch. Her current research examines fundamental mechanisms that regulate gene expression, focusing on the function of specific heterogenous nuclear ribonucleoproteins in the regulation of RNA biosynthesis and the discovery of cancer-specific dependencies, with a concentration on the study of EWSR1-fusion driven tumors. In February 2025, Dr. Caplen was awarded tenure and appointed to Senior Investigator.