Christina A. Pacak, Ph.D. is a molecular biologist whose research focuses on uncovering the mechanisms underlying rare disorders to inform the development of therapies relevant to both rare and common diseases. The laboratory has found intriguing links between hallmarks of aging and mitochondrial dysfunction across multiple rare diseases. The laboratory uses patient-derived induced pluripotent stem cells (iPSCs), mouse models, and large animal models to investigate disease biology. In parallel, the lab develops and optimizes adeno-associated virus (AAV)–mediated gene delivery systems, with ongoing efforts to advance treatments for the accelerated aging disease Cockayne syndrome and other inherited genetic disorders.