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Welcome to the Gomez-Ospina Lab

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About the Gomez-Ospina Lab

Precise Genetic Medicines

The main focus of Dr. Gomez-Ospina’s lab is to develop therapies for patients with genetic diseases. The lab uses genome editing and stem cells to produce definitive treatments for childhood neurodegenerative diseases, many of which are lysosomal storage disorders. 

Current projects in the lab include developing autologous transplantation of genome-edited hematopoietic stem cells for Mucopolysaccharidosis type I, Gaucher, Krabbe disease, Frontotemporal Dementia, and Friedreich's ataxia.


Although there is a strong translational focus to the lab, we are also pursuing basic science questions to understand and enhance our therapies including

  1. increasing the efficiency of genome editing tools,

  2. understanding microglia turnover in response to conditioning before hematopoietic stem transplant, and

  3. establishing brain-specific conditioning regimens to neurometabolic diseases.

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Dr. Natalia Gomez-Ospina


Dr. Gomez-Ospina was born and raised in Medellin, Colombia. She began her undergraduate studies in petroleum engineering at the Universidad Nacional de Colombia before moving to Colorado. She double majored at the University of Colorado Boulder, completing her bachelor’s degree in Molecular Cellular and Developmental Biology and Biochemistry. She graduated summa cum laude and wrote an honors thesis entitled “Role of the quiescent center in the regeneration of the root cap in Zea Mays.” She then completed her combined MD, Ph.D., at Stanford Medical School, where her Ph.D. work focused on understanding the novel functions of voltage-gated calcium channels.  After completion of her dual degrees, she did her preliminary year in internal medicine at Santa Barbara Cottage hospital before starting a residency in Dermatology at Johns Hopkins Hospital. She completed a residency in Medical Genetics at Stanford which was followed by post-doctoral research in Pediatric Stem Cell transplantation. As a post-doc, she began the development of a genome editing strategy in stem cells as a curative therapy for metabolic diseases. Her current research aims to develop treatments for patients with genetic disorders with a particular focus on lysosomal storage diseases.


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.

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Our lab is currently welcoming students, post-docs, and research assistants. Contact us for details, 650-725-9708

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