Dr. Gomez-Ospina is an Assistant Professor in the Department of Pediatrics at Stanford University. She is a board-certified medical geneticist interested in developing definitive therapies for individuals with genetic diseases. She leads a Program for Inherited Metabolic Diseases at Stanford to promote gene and cell-based therapies for metabolic diseases. She has been the lead author in research studies in The New England Journal of Medicine, Cell, Nature Communications, and American Journal of Medical Genetics.

Dr. Gomez-Ospina's research program is focused on developing genome editing in hematopoietic stem cells as a therapeutic approach for lysosomal storage disorders. She established a precise genetic approach where lysosomal enzymes, or other therapeutic proteins, can be targeted to a single well-characterized place in the genome known as a safe harbor. This approach constitutes a flexible, "one-size-fits-many"strategy independent of specific genes and mutations and can be used to engineer any human cell. When applied to hematopoietic stem cells, these cells are recruited to express and deliver lysosomal enzymes to the brain. This approach was effective in preclinical models Mucopolysaccharidosis type I (MPSI or Hurler syndrome) and Gaucher disease and is being developed for Krabbe and Progranulin deficiency.