Genomic mechanisms of human myelopoiesis: implications for bone marrow reconstitution after gene therapy

A rapid regeneration of the immune system after hematopoietic stem cell (HSC) transplantation is key for the safety and efficacy of gene therapy. In this project, we employ advanced technologies to dissect the reconstitution of neutrophils and monocytes - cell type with critical roles in protecting against infections - in patients undergoing HSC transplant. Our studies are highly innovative as they apply complex genomic and computational analyses to the study of clinically relevant material. These data will highlight novel biomarkers or therapeutic targets to further increase the safety and efficacy of HSC transplantation, with broad implications for gene therapy approaches for the treatment of genetic disease.