External Resources:

Stephanie Cherqui, Ph.D.

Associate Professor of Pediatrics, Division of Genetics

Website: https://medschool.ucsd.edu/som/pediatrics/research/labs/cherqui/Pages/default.aspx
Email: scherqui@health.ucsd.edu

Dr. Cherqui’s laboratory specialized in the translation of gene therapy strategy from Bench-to-Bedside. She established that hematopoietic stem and progenitor cells (HSPCs) transplantation could reverse multi-organ degenerative disorders, even in the context of an intracellular membrane protein such as cystinosis. Her work led to the first in human HSPC gene therapy clinical trial for cystinosis. She is now applying this strategy to other lysosomal and mitochondrial genetic disorders.