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.jpgDr. Cherqui received her Ph.D in Human Genetics from the University Rene Descartes (Paris, France) in 2002. Her research project focused on the lysosomal storage disorder cystinosis and the identification of the gene involved in this disease, its function and the generation of a mouse model for cystinosis.

Dr. Cherqui then specialized in stem cells and gene therapy applied to vascular diseases during her post-doctoral internship at The Scripps Research Institute.​ Read more here 


The work performed in Dr. Cherqui’s lab represents the first proof of concept for using HSPC transplantation as a therapy for cystinosis and led to the first stem cell clinical trial for this disease. The IRB for an allogeneic transplantation protocol has been approved at the University of California Los Angeles in April 19th, 2012.

Read more here.

Sacramento Assemblyman, Dr Richard Pan, a member of the California State Health committee, awarded Dr. Cherqui with a State Resolution for her exceptional and tireless work on behalf of the cystinosis community.