Stefan Karlsson – ULUND – University of Lund, Lund, Sweden

stefankarlssonInstitute description
The Department of Molecular Medicine and Gene Therapy of the University of Lund consists of approximately 20 scientists, eleven of whom work for Prof. Karlsson.

The research laboratory has a long-standing experience, spanning two decades, on the regulation of hematopoietic stem cells and on how to develop gene therapy for hematopoietic stem cells.
The main expertise of the laboratory involves the development of transgenic and knock-out mouse models, hematopoiesis research including the purification of hematopoietic stem cells and bone marrow transplantation studies, and vector development for gene therapy of hematological disorders.

One of the research projects involves the development of animal models and gene therapy for Gaucher disease. The Lund University Strategic Center for Stem Cell Biology and Cell Therapy has recently developed a mouse model for Gaucher disease that is crucial for the gene therapy studies proposed in the EUCLYD project (WP5). Furthermore, this model will be shared with other investigators within the consortium to evaluate SRT for Gaucher disease (WP4).

Group leader
Stefan Karlsson, MD, is Full Professor of Molecular Medicine and Senior Physician at Lund University Hospital since 1995. Prof. Karlsson was formerly the Head of the Molecular and Medical Genetics Section, NINDS, NIH, Bethesda, 1986-1996. His main expertise involves hematopoiesis, hematopoietic stem cells and development of gene therapy for hematopoietic stem cells.

Role in the project
He is involved in developing safe gene therapy for Gaucher disease using a novel animal model developed within the laboratory (WP5) and to share this model with other investigators within the consortium to evaluate substrate reduction therapy for Gaucher disease (WP4).

Key personnel

Name Title Gender Role/Expertise
Stefan Karlsson Group leader M Involved in WP4, WP5
Johan Richter Investigator M Hematology aspects of the project
Ida-Berglin Enquist PhD Student F Generation of mouse models and gene therapy for Gaucher disease
Eva Nilsson Senior Technician F Generation of mouse models and gene therapy for Gaucher disease
to be appointed PhD Student

Key publications

Enquist IB, Nilsson E, Ooka A, Mansson JE, Olsson K, Ehinger M, Brady RO, Richter J, Karlsson S (2006). Effective cell and gene therapy in a murine model of Gaucher disease. Proc Natl Acad Sci USA 103: 13819-13824.

Woods NB, Muessig A, Schmidt M, Flygare J, Olsson K, Salmon P, Trono D, von Kalle C, Karlsson S (2003). Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis. Blood 101: 1284-1289.

Hamaguchi I, Flygare J, Nishiura H, Brun AC, Ooka A, Kiefer T, Ma Z, Dahl N, Richter J, Karlsson S (2003). Proliferation deficiency of multipotent hematopoietic progenitors in ribosomal protein S19 (RPS19)-deficient diamond-Blackfan anemia improves following RPS19 gene transfer. Mol Ther 7: 613-622.

Hamaguchi I, Ooka A, Brun A, Richter J, Dahl N, Karlsson S (2002). Gene transfer improves erythroid development in ribosomal protein S19-deficient Diamond-Blackfan anemia. Blood 100: 2724-2731.

Mikkola H, Woods NB, Sjogren M, Helgadottir H, Hamaguchi I, Jacobsen SE, Trono D, Karlsson S (2000). Lentivirus gene transfer in murine hematopoietic progenitor cells is compromised by a delay in proviral integration and results in transduction mosaicism and heterogeneous gene expression in progeny cells. J Virol 74: 11911-11918.