The Division of Hematology, Oncology, & Transplantation has identified three diseases: sickle cell disease (SCD), Fanconi anemia (FA) and primary immunodeficiency (PID) for which we will use targeted genome engineering tools to correct the endogenous mutated locus. We hypothesize that efficient, site-specific editing of endogenous loci in large numbers of autologous HSC will enhance the efficacy of current genetic engineering. Each project addresses unique challenges in the gene therapy field, specifically, safe delivery, gene targeting efficiency, adverse off-target effects, absolute number of gene-modified HSC, and a plethora of host factors including immune response to gene modified cells and residual host HSC repopulation. The goal will be to achieve high-efficiency genetic modification with correction of biological function. A long-term goal is to develop gene editing delivery in vivo in murine models and ultimately in humans.

This position will work with Drs. Vercellotti, Wagner, Belcher, Moriarity, Webber, and McIvor and will be assigned two or more research projects in which the individual will be responsible for progress based on the following:

40% Design and conduct experiments and maintain excellent, detailed documentation.

30% Analyze and interpret data, prepare figures for manuscripts and presentations, maintain expertise in the relevant literature. Communicate and meet with PIs.

20% Write manuscripts, progress reports, abstracts, assist with grant- writing.

5% Present data at lab meetings, local and international conferences.

5% Assist in laboratory management, inventory, ordering, organization, and other miscellaneous lab duties.

See attached for full position and application info.