
Our laboratory focuses on developing stem cell and gene therapy strategies for degenerative multi-systemic disorders such as cystinosis, and to understand the molecular mechanisms by which hematopoietic stem cell progeny can lead to tissue repair in non-hematopoietic genetic diseases.
Meet the PI
Dr. Cherqui is a professor at the University of California San Diego, Department of Pediatrics. She received her Ph.D in 2002 at Necker hospital (Paris, France); her research project focused on the molecular characterization of cystinosis, a metabolic hereditary disease, as well as the generation of mouse models. 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, became an Associate Professor in 2016, and Professor in 2022.
News
Novartis Deal Boosts CIRM Approach The therapy was sold for $87.5 million by Avrobio, Inc., of Cambridge, Ma., to Novartis AG, a $52-billion-a-year firm based in Switzerland. The treatment was developed by Stephanie Cherqui of UC San Diego.
Best Life: Experimental treatment for cycstnosis The Department of Pediatrics is excited to share this news clip that highlights Dr. Stephanie Cherqui’s experimental gene therapy approach to treating cystinosis. Stem cells taken from patient’s peripheral blood were re-engineered to produce functional cystinosin, the protein defective in cystinosis. The patient was then reinfused with his own cystinosin-producing cells.
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