Natalia Gomez-Ospina is an Assistant Professor of Pediatrics (Genetics) and of Pediatrics (Stem Cell Transplantation) at Stanford University. Dr. Gomez-Ospina was born and raised in Medellin, Colombia. Her current research aims to develop treatments for patients with genetic disorders with a particular focus on lysosomal storage diseases including Mucopolysaccharidoses (MPS).

Dr. Gomez-Ospina completed her combined MD, PhD at Stanford Medical School. Her post-doctoral research was with Dr. Matthew Porteus in Pediatric Stem Cell transplantation, where she began to develop genome editing-based strategies in stem cells as therapy for metabolic diseases. For her clinical practice she sees patients with suspected genetic disorders, and is also in charge of the enzyme replacement service for lysosomal storage disorders at Lucile Packard Children’s hospital. 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.

As a board-certified Medical Geneticist., Dr. Gomez-Ospina’s clinical responsibilities include the clinical evaluation and diagnosis of patients with suspected genetic conditions. She also manages the treatment of some of these diseases at Stanford by overseeing the enzyme replacement service and leading the Program for Inherited Metabolic Diseases (PIMD), which aims to promote the preclinical development of gene and cell-based therapy approaches for metabolic diseases.

Representative Publications:

Human Genome-Edited Hematopoietic Stem Cells Phenotypically Correct Mucopolysaccharidosis Type I

Genome Editing for Mucopolysaccharidoses

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