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The main focus of Dr. Cherqui’s lab is to develop stem cell and gene therapy strategies for degenerative multi-systemic genetic disorders, and to understand the mechanisms by which hematopoietic stem cells can lead to tissue repair. Her research led to the first autologous hematopoietic stem cell gene therapy clinical trial for cystinosis, a lysosomal storage disorder. Dr. Cherqui received her PhD in 2002, and was appointed Assistant Professor in 2009 at the Scripps Research Institute. In 2012, she joined the University of California San Diego in the Department of Pediatrics, and became Associate Professor in 2016. Dr. Cherqui is the chair of the Cystinosis Stem Cell and Gene Therapy Consortium. She is also co-founder of GenStem Therapeutics, Inc. Her research is funded by grants from the National Institute of Health (NIH), California Institute of Regenerative Medicine (CIRM), and the Cystinosis Research Foundation.
Hematopoietic Stem Cell Gene Therapy For Cystinosis
This presentation will provide an overview of the steps necessary to go from Bench to Bedside for an autolo-gous transplantation of gene-corrected HSPCs using a SIN-lentiviral vector for cystinosis, a lysosomal storage disorder that leads to multi-organ failure. We performed the toxicology studies and manufacturing develop-ment, and recently started a phase I/II clinical trial at UCSD.