We study the regulation of hematopoietic stem cells in order to understand how signaling pathways, transcriptional mechanisms govern stem cell behavior to determine stem cell fate options. Integrative signaling and transcriptional networks act in concert to determine whether hematopoietic stem cells remain quiescent, proliferate and self-renew, expand through self-renewal, differentiate or undergo apoptosis. We study various regulators to determine how we can 1) expand stem cells 2) understand the pathogenesis of blood malignancies and genetic disorders of the blood system and 3) develop cell and gene therapies. Goals • Develop methods to expand hematopoietic stem cells and early progenitors to improve engraftment following blood and marrow transplantation. • Understand the pathogenesis of Diamond Blackfan anemia and develop mechanism-based therapies for this disorder, including gene therapy. • Develop curative gene therapy for the most common lysosomal storage disorder, Gaucher disease.

Stefan Karlsson is Research Group Leader for this research group