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 


Cystinosis Research Foundation grants support to various scientific studies on Cell Function, New Treatments and the Quest for a Cure. $2.79 Million Awarded in 2016 to Fund 17 Research Projects in Five Countries. Among 9 funded projects in the US is the "Accelerated Research Program for Stem Cell Research" of Stéphanie Cherqui, Ph.D.

"Our work in the area of nanotechnology and corneal cystinosis will undoubtedly help other corneal diseases. The stem cell work conducted by Stéphanie Cherqui, Ph.D., (UCSD) will help treat other lysosomal and even neuro-muscular degenerative disorders. CRF research extends far beyond our community; it has the potential to treat millions of other people who suffer from other disorders." Latest News