About the Lab
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 received her Ph.D in 2002 at Necker hospital (Paris, France); her research project focused on the molecular characteri-zation 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 and became an Associate Professor in 2016.
Stephanie Cherqui and Natalie's Wish: Finding a Gene Therapy for Cystinosis. The story of what has driven UC San Diego's Stephanie Cherqui to develop a gene therapy for Cystinosis, a devastating inherited condition.
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.
CIRM Board Approves New Clinical Trial for Rare Childhood Disease The governing Board of the California Institute for Regenerative Medicine (CIRM) approved a grant of almost $12 million to Dr. Stephanie Cherqui at the University of California, San Diego (UCSD) to conduct a clinical trial for treatment of cystinosis.
Wed May 15 Dr. Stephanie Cherqui was honored today with the Rady Children's Hospital-San Diego Scientist Award of Excellence Award for her research work on cystinosis.
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