The main focus of her lab is to develop stem cell and gene therapy strategies for degenerative multi-systemic disorders, and to understand the mechanisms by which hematopoietic stem and progenitor cells (HSPC) can lead to tissue repair. Her work is leading to the first-in-human clinical trial for autologous hematopoietic stem cell gene therapy for cystinosis, but also her findings on the cellular mechanism of HSPC-mediated tissue preservation led to the application of this strategy to other disorders including the neuro-muscular degenerative disease, Friedreich’s ataxia.
Stephanie Cherqui received her Ph.D in 2002 at Necker hospital (Paris, France); her research project focused on the molecular characterization of cystinosis and the generation of the mouse model. She then specialized in stem cells and gene therapy during her post-doctoral internship at The Scripps Research Institute where she was appointed Assistant Professor in 2009. In 2012, Dr. Cherqui joined the University of California, San Diego, Department of Pediatrics, Division of Genetics and became Associate Professor in 2016.
She is the Chair of the Cystinosis Stem Cell and Gene Therapy Consortium. Dr. Cherqui is also the chair of the Gene and Cell Therapy of Genetic and Metabolic Diseases at the American Society of Gene and Cell Therapy (ASCGT). She is 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 funded by grants from the National Institute of Health (NIH), California Institute of Regenerative Medicine (CIRM), and the Cystinosis Research Foundation.