About the Lab

‚ÄčOur 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 


A Steady Increase in Women Leading Biggest Research Grants. Six of Top 10 research health science grants awarded to UC San Diego this year are led by women.

Transplant Hematopoietic Stem Cells Reverse Damage Caused by Neuro-Muscular Disorder. The findings, published in Science Translational Medicine, suggest one potential approach for a disease that is considered incurable. In their study, Dr. Stephanie Cherqui and colleagues used a transgenic mouse model that expresses two mutant human FXN transgenes...

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