Decades of biological research have unveiled the genetic causes of many debilitating diseases. This knowledge unlocks the promise and potential of gene therapy: a drug administered one time that can modify the genetic information in a patient’s cells. This mechanism makes it possible to provide treatments that were once only imagined. Depending on the disease, scientists can use different strategies to target the genetic trigger. For example, scientists can genetically modify a patient’s chromosome to either introduce a healthy copy of a gene into a patient’s cells or shut off the mutant gene. Gene therapies have the potential to one day dramatically change how humans respond to disease, moving from disease management to transformative medicines that may potentially prevent, halt, or cure a disease with one injection.
For Junghae, pausing to read that poster on the wall years ago marked the beginning of her journey in the field of gene therapy, leading her to discover a new approach to track and measure the way gene therapies move within live cells.1
For years, researchers worked to understand what stopped gene therapies from reaching the nucleus of a cell and delivering their therapeutic package. Using Junghae’s approach, researchers could see a real-time movie of the gene therapy in cells, instead of just snapshots. By seeing particles travel through cells, Junghae determined that the gene therapy was stuck on the intracellular highway with no exit ramp to the nucleus.
By understanding the problem, Junghae could focus on a solution. She looked to nature for inspiration, pursuing a postdoctoral research fellowship in a fundamental virology laboratory at the Salk Institute for Biological Studies.