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,...
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