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 P.I.

Stephanie Cherqui, Ph.D.jpg 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. Read more 


AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Cystinosis.  Avrobio, Inc. is a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases. “This cystinosis program is a strong strategic fit... We are pleased to partner with Dr. Stephanie Cherqui and her team at the University of San Diego (UCSD)," said Geoff MacKay, AVROBIO’s President and CEO.

A life-threatening childhood disease and CIRM-funded team seeking the stem cell cure.  Such cure is Clay Emerson’s biggest hope for his young daughter Brooke, who has cystinosis, a genetic disease that over time causes damage to many organs. UCSD researcher Dr. Stephanie Cherqui, received $5 million in CIRM funding to bring her team’s therapy to clinical trials in people.

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