School of Medicine
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Director of Finance and Administration (SoM), Stem Cell Bio Regenerative Med Institute
Current Role at StanfordI am the Director of Finance and Administration for the Institute for Stem Cell Biology and Regenerative Medicine (ISCBRM) in the School of Medicine. I oversee and carry out administrative and financial related functions for the department.
Assistant Professor of Pediatrics (Genetics) and of Pediatrics (Stem Cell Transplantation)
Current Research and Scholarly InterestsDr. Gomez-Ospina is a physician scientist and medical geneticist with a strong interest in the diagnosis and management of genetic diseases.
1) Lysosomal storage diseases:
Her research program is on developing better therapies for a large class of neurodegenerative diseases in children known as lysosomal storage disorders. Her current focus is on developing genome editing of hematopoietic stem cells as a therapeutic approach for these diseases beginning with Mucopolysaccharidosis type 1 and Gaucher disease. She established a genetic approach where 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 all” approach that is independent of specific genes and mutations. This strategy, in which the hematopoietic system is commandeered to express and deliver therapeutic proteins to the brain can potentially change the current approaches to treating childhood neurodegenerative diseases and pave the way for alternative therapies for adult neurodegenerative disorders such as Alzheimer’s and Parkinson’s disease
2) Point of care ammonia testing
She also works in collaboration with other researchers at Stanford to develop point-of-care testing for serum ammonia levels. Such device will greatly improve the quality of life of children and families with metabolic disorders with hyperammonemia.
3) Gene discovery
Dr Gomez-Ospina lead a multi-institutional collaboration resulting in the discovery of a novel genetic cause of neonatal and infantile cholestatic liver disease. She collaborated in the description of two novel neurologic syndromes caused by mutations in DYRK1 and CHD4.
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Postdoctoral Scholar, Stem Cell Biology and Regenerative Medicine
BioI am a zoologist and molecular biologist interested in the molecular basis of regeneration. My research focuses on stem cells and regeneration in ascidians, a group of marine invertebrates that represent the closest living relatives of the vertebrates. One of the main questions that motivate my research is whether regeneration capabilities lost during evolution can, at least to some extent, be re-acquired. As regeneration is not universal in the animal kingdom, I hypothesize that comparing regeneration in species with distinct regenerative capacities will lead to the discovery of key components of regeneration.
During my postdoc I intend to use comparative genomics to identify conserved cellular and molecular mechanisms that underlie ascidians’ regeneration.