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 


​'A Tornado at the Front Door, a Tsunami at the Back Door'  The first cystinosis patient undergo new gene-and-stem cell therapy developed by School of Medicine researchers.

AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis The first cystinosis patient was transplanted on October 7, 2019, a day that will be forever remembered as a historic day for adults and children with cystinosis and their families. 

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.

FDA Approved First Human Clinical Trials for Cystinosis The Cystinosis Research Foundation (CRF), the largest provider of grants for cystinosis research in the world, today announced that the Federal Drug Administration granted approval for the first human clinical trial for stem cell and gene therapy treatment for cystinosis. Over the last 12 years, CRF has provided more than $4.3 million in research funding to Dr. Cherqui. She will be leading the trial at UC San Diego in San Diego, California...

CRF Announces FDA Approval for First Human Clinical Trials The world's largest provider of grants for cystinosis research announced the Federal Drug Administration's approval for first human clinical trial regarding stem cell and gene therapy treatment for cystinosis...

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