ASCI / Young Physician-Scientist Awards, 2021
The Young Physician-Scientist Awards (YPSA) recognize physician-scientists who are early in their first faculty appointment and have made notable achievements in their research.
About the awardee
Natalia Gomez-Ospina, MD, PhD, is a physician-scientist dedicated to the diagnosis and treatment of genetic diseases. Her clinical interest focuses on patients with lysosomal storage disorders (LSDs), the most common genetic cause of childhood neurodegeneration. She directs the enzyme replacement service for LDSs at Lucile Packard Children’s hospital and the Program for Inherited Metabolic Disorders at Stanford, which aims to promote gene and cell-based therapies for LSDs. Dr. Gomez-Ospina has championed the idea of commandeering the hematopoietic system to express proteins needed in other organs, including the brain. She established an adaptable platform for the treatment of lysosomal enzyme deficiencies and performed a first-of-its-kind preclinical study to support the clinical development of autologous transplantation of genome edited cells to treat patients with Mucopolysaccharidosis type I (Hurler syndrome). Beyond delivering lysosomal enzymes, this platform has potential implications for delivering many kinds of therapeutic proteins to the brain. Her lab combines basic and translational science needed to develop therapies for LSDs primarily combining stem cells and genome editing. In addition to therapy development, Dr. Gomez-Ospina led the discovery and characterization of a new infantile cholestatis syndrome caused by mutations in the bile acid receptor and, as part of large multi-institutional collaborations, participated in the discovery of multiple genetic syndromes.