School of Medicine
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MD Student, expected graduation Spring 2023
Ph.D. Student in Stem Cell Biology and Regenerative Medicine, admitted Summer 2017
BioMy journey to pursue the physician-scientist track stems from an early fascination with biology and my family’s eight-year struggle to save my younger brother’s life. My brother was born with a complex congenital heart defect known as Hypoplastic Left Heart Syndrome (HLHS). In 2003, my brother received a heart transplant, but despite this, he passed away in 2004. Growing up, I strived to find an explanation for my brother’s congenital heart defect and became interested in medicine at a young age. Looking to pursue this goal, I attended the Michael DeBakey High School for Health Professions, a top pre-health and science public school, and in 2011 I was accepted to Harvard University where I pursued a major in the department of Stem Cell and Regenerative Biology. During my college years I took classes in developmental and stem cell biology where I became interested in the use of these fields to understand congenital defects. I began my research career in Drs. Caroline and Geoffrey Burn’s lab studying the development of the great vessels of the heart in zebrafish, Throughout my time at Harvard, I was awarded numerous fellowships that supported my undergraduate research, including the prestigious Amgen Scholars Fellowship that supported summer research in Dr. Michael Longaker’s lab at Stanford University. I pursued my undergraduate honors thesis in Dr. Richard Lee’s laboratory where I identified the role of Apolipoprotein E as a factor necessary for maintaining mature beta cell gene expression. These experiences culminated in my decision to apply to the physician-scientist program at Stanford University where I am currently training to pursue a career in academic medicine with the ultimate goal of practicing as a pediatric cardiologist and a university professor with an active laboratory. Currently, I am a graduate student in Dr. Sean Wu’s laboratory where I study the development of ventricular development in the heart using both bioinformatic approaches such as scRNA-seq and human induced pluripotent stem cells to study the development of the left and right ventricles. Through my work, I aim to understand the mechanisms that give rise to single ventricle congenital heart defects with the hope of making a difference in the lives of children born with these diseases.
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.