​​ 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

Stephanie Cherqui, Ph.D.jpg

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.


Stem Cell Therapy Rescues Symptoms of Alzheimer’s Disease UC San Diego study shows hematopoietic stem cell transplants can protect against memory loss, neuroinflammation and β-amyloid build-up in Alzheimer’s mice. This new proof-of-concept study shows stem cell transplants may also be a promising therapeutic against Alzheimer’s​.

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.

Beaker List - Top 23 Female Academic Entrepreneurs In honor of International Women's Day 2023, BIOS community have put together the "Top 23 Women in Academic Entrepreneurship" that have changed the life sciences ecosystem.

Scientists Receive $4.8M to Pursue Gene Therapy for 'Incurable' Disease CIRM grant will fund novel gene therapy aiming for one-time, lifelong treatment of Friedreich’s ataxia, a progressive neuromuscular disorder; second CIRM grant will advance efforts to leverage UC San Diego research on another rare disease

Stephanie Cherqui and Natalie's Wish: Finding a Gene Therapy for CystinosisThe story of what has driven UC San Diego's Stephanie Cherqui to develop a gene therapy for Cystinosis, a devastating inherited condition.


Side Effects May Include … A Completely New Hair Color? An experimental therapy helped patients with a rare disease feel better. It also led to an accidental makeover.

Best Life: Experimental treatment for cycstnosisThe 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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