Hematopoiesis and Gene Therapy

Organisational unit: Research group

Research areas and keywords

UKÄ subject classification

  • Cell and Molecular Biology


  • Hematopoiesis, hematopoietic stem cells, gene therapy


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.

Recent research outputs

Stefan Karlsson, 2018 Jul 19, In : New England Journal of Medicine. 379, 3, p. 295-296 2 p.

Research output: Contribution to journalDebate/Note/Editorial

Rajendran, V. & Mayur Vilas Jain, 2018, Methods in Molecular Biology. Humana Press, Vol. 1692, p. 89-95 7 p. (Methods in Molecular Biology; vol. 1692)

Research output: Chapter in Book/Report/Conference proceedingBook chapter

Komorowska, K., Alexander Doyle, Martin Wahlestedt, Agatheeswaran Subramaniam, Debnath, S., Chen, J., Shamit Soneji, Van Handel, B., Mikkola, H. K. A., Kenichi Miharada, David Bryder, Jonas Larsson & Mattias Magnusson, 2017 Dec 19, In : Cell Reports. 21, 12, p. 3514-3523 10 p.

Research output: Contribution to journalArticle

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