Institute for Stem Cell Biology and Regenerative Medicine
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Heather Gentner
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.
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Natalia Gomez-Ospina
Assistant Professor of Pediatrics (Genetics)
Current Research and Scholarly Interests1. Genome Editing and HSPC-Based Therapeutics
Dr. Gomez-Ospina’s research focuses on developing autologous hematopoietic stem and progenitor cell therapies using CRISPR/Cas9-mediated genome editing. Her work includes targeted integration of therapeutic transgenes into genomic safe-harbor loci, optimization of editing efficiency and safety, and development of transplantation and conditioning strategies that preserve long-term engraftment. A major goal is to create reusable, mutation-agnostic therapeutic platforms that can be adapted across diseases. Her translational work also extends to GMP manufacturing, analytical development, and IND-enabling studies needed to move genome-edited HSPC products toward clinical trials.
2. Neurometabolic and Neurodegenerative Disease Therapeutics
A major area of interest is the development of definitive therapies for lysosomal storage disorders and other neurogenetic diseases, including MPS I, Gaucher disease, Krabbe disease, progranulin deficiency, and Friedreich’s ataxia. Her laboratory investigates how hematopoietic-derived cells can deliver therapeutic proteins to peripheral tissues and the central nervous system. This includes strategies to enhance microglial replacement, improve CNS engraftment, and harness myeloid cells as therapeutic vehicles. The broader objective is to establish cell-based approaches capable of treating both inherited neurometabolic disorders and more common neurodegenerative diseases.
3. Rare Disease Genetics, Functional Genomics, and Lysosome Biology
Dr. Gomez-Ospina also studies the molecular basis of rare and undiagnosed genetic disorders. Through clinical genetics, international collaborations, and the NIH Undiagnosed Diseases Network, her work contributes to novel disease-gene discovery, functional validation of candidate variants, and definition of genotype–phenotype relationships. Her laboratory also investigates lysosomal biology and develops systems-level approaches, including the Lysosome Disease Atlas, to connect lysosome-associated genes with cellular function and human phenotypes. These efforts aim to improve diagnosis while identifying new disease mechanisms and therapeutic opportunities.
For more information go to our website:
https://www.gomezospina.com/
