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Stephanie Cherqui, PhD

Professor, Department of Pediatrics, University of California San Diego


Stephanie Cherqui is Professor in the Department of Pediatrics, Division of Genetics at the University of California San Diego. The main focus of her laboratory is the development of stem cell and gene therapy-based therapeutic approaches for genetic disorders, and the understanding of the mechanism of action for hematopoietic stem cell-mediated tissue repair. She established that hematopoietic stem and progenitor cell (HSPC) transplantation could reverse multi-organ degenerative disorders, even in the context of an intracellular membrane protein such as cystinosis, a lysosomal storage disorder. Her work led to the first in human HSPC gene therapy clinical trial for cystinosis. She is now applying this strategy to other disorders including the neuro-muscular degenerative disease, Friedreich’s ataxia. Dr. Cherqui received her Ph.D in 2002 at Necker hospital (Paris, France) and was appointed Assistant Professor at The Scripps Research Institute in 2009. She joined UC San Diego in 2012. She is the Chair of the Cystinosis Stem Cell and Gene Therapy Consortium, and a member of the American Society of Gene and Cell Therapy (ASCGT) Gene and Cell Therapy of Genetic and Metabolic Diseases committee. She is also a member of the Scientific Review Board of the Cystinosis Research Foundation and a Scientific Council member for the Cure Cystinosis International Registry (CCIR). Her research is mainly funded by grants from the National Institute of Health (NIH), California Institute of Regenerative Medicine (CIRM), the Cystinosis Research Foundation, and Friedreich’s ataxia Research Alliance (FARA).


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