Institute for Stem Cell Biology and Regenerative Medicine
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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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Gunsagar S. Gulati
Affiliate, Stem Cell Bio Regenerative Med Institute
BioI am an aspiring physician-scientist interested in the crossroads of computer science, cellular biology, and clinical medicine. I am particularly motivated to leverage recent advances in single-cell genomics and epigenomics to study normal and neoplastic stem cell biology, with the ultimate goal of addressing human cancer.
Postdoctoral Scholar, Stem Cell Biology and Regenerative Medicine
BioDr. Gurjar is currently developing a small molecule inhibitor for the treatment of triple negative breast cancer at Stanford ChEM-H Medicinal Chemistry Knowledge Center with Drs. Mark Smith and Michal Clarke. Prior to joining Stanford, he obtained his PhD in Chemistry from the lab of Prof. Valery Fokin, founder of copper-catalyzed azide-alkyne (CuAAC) click chemistry, at the University of Southern California. His research focused on developing new click reactions and their applications spanning across small molecule inhibitors to materials chemistry.