Postdoctoral Fellow – Stem Cells Gene Therapy/Gene editing for inherited disorders- Univ of California

Applicants are invited for a postdoctoral fellowship in the Division of Genetics of the Department of Pediatrics at the University of California San Diego. The Department of Pediatrics is one of the largest departments within the School of Medicine. The missions of research, education, and patient care are intertwined and are integral to the goals of the department.

Cherqui laboratory focuses on developing stem cell and gene therapy strategies for degenerative multi-organ disorders, and to understand the molecular mechanisms by which hematopoietic stem cell progeny can lead to tissue repair in genetic diseases. Our work led to a the first in human clinical trial for autologous transplantation of gene-corrected hematopoietic stem cells for cystinosis, a lysosomal storage disorder, but also to the application of this strategy to other disorders including a neuro-muscular degenerative disease, Friedreich’s ataxia.

Lab/Research Program Website

Job Title

Postdoctoral Fellow – Stem Cells Gene Therapy/Gene editing for inherited disorders



University of California, San Diego

Department of Pediatrics


Principal Investigator

Stephanie Cherqui, PhD


Disciplinary Specialty of Research

Gene therapy, Gene editing, Stem Cells, Neuroscience


Description of the Position

Available immediately for highly motivated Ph.Ds, the position focuses on the use of hematopoietic stem cells (HSCs) and gene therapy for the treatment of inherited approaches for multi-systemic degenerative disorders, and in particular neuromuscular diseases. Gene therapy technologies employed in the project include lentivirus vectors and CRISPR/Cas9-mediated gene editing. The candidate will also use human and mouse HSCs in in vitro assays and murine models of genetic disorders. The project also includes the investigation of the mechanism involved in the rescue of degenerative neuromuscular tissues by HSC-derived cells. Successful Candidates will be part of a stimulating and collaborative scientific environment with cutting-edge instrumentation and facilities.

Qualifications Required and Preferred Academic Background

The candidate should be highly self-motivated, with strong communication and interpersonal skills and the ability to work independently, has a PhD. Experience with mice is required. Background in stem cell biology, gene therapy, CRISPR/Cas9-mediated gene editing or neuroscience is preferred.



Salary is commensurate with experience.


Appointment Length/Period

Two years, with possibility for extension.


How to Apply?

Review of applications will begin immediately and continue until the position is filled. If you are interested, please submit your C.V. to Stephanie Cherqui


*All qualified applicants will receive consideration for employment without regard to race, color, religion, sex, sexual orientation, gender identity, national origin, disability, or status as a protected veteran.