Stephanie Cherqui, PhD

University of California, San Diego, Associate Professor

What is your current professional status?

Associate Professor

What is your work setting (i.e. academic institution, government organization (i.e. FDA, NIH, etc.), bio-industry/pharmaceutical company, etc.)?

University of California, San Diego

What is your scientific area of research?

Hematopoietic stem cells, Gene Therapy, Lentivirus Vectors, CRISPR-Cas9-mediated gene editing

Why do you want to be a mentor?

Along my career, I was inspired by mentors who took the time to explain their research and to advise me on the best path to follow to reach my goals. I now want to help the young scientists who have many different opportunities but also challenges to overcome. I am an Academic Researcher, but I also work closely with industry and created my own company, so I can provide advises in both career plans.

As a mentor, what are you hoping to gain from this experience?

It is rewarding to meet young, motivated researchers in the field of stem cell and gene therapy and to have the opportunity to advise them.

How important are the following in the selection of a mentee: (1 – 5 scale, 1 = most important, 5 = least important)

  • Compatibility of Scientific Specialties: 2
  • Geographic Location: 5
  • Professional Status / Success: 3
  • Compatibility of Career Path: 1 (most)
  • Communication Style: 4

Summarize in a brief paragraph (150 words or less), please provide a biography that describes who you are and what you do in the field of gene and/or cell therapy:

The main focus of my lab is to develop stem cell and gene therapy strategies for degenerative multi-systemic disorders, and to understand the mechanisms by which hematopoietic stem and progenitor cells (HSPCs) can lead to tissue repair. My lab is set to go from Bench to Bedside and is now leading to the first-in-human clinical trial for autologous hematopoietic stem cell gene therapy for cystinosis. Our findings on the cellular mechanism of HSPC-mediated tissue preservation led to the application of this strategy to other disorders for which are also developing the clinical translation of ex vivo gene therapy approaches.

View CV


23rd Annual Meeting

May 12-15 | Boston