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Our SCIDNET proposal to cure Severe Combined Immunodeficiency (SCID) to in the context of the European Union Horizon2020 scheme is selected for funding. This grant allows us to work on SCID treatment with European leaders in field of gene therapy.
For more information see the press release (in Dutch).

Elsevier, a current opinions magazine in the Netherlands, interviewed Frank Staal about Pompe's disease, cystic fibrosis and XSCID. (article in Dutch)

Dutch popular science radio show "Labyrint" interviewed Frank Staal and Sander van Deventer about new developments in gene therapy, for example about Glibera and SCID.
(interview in Dutch, starting at 29 min.)


The Molecular Stem Cells Biology laboratory at the Leiden Universtity Medical Center is headed by professor Frank Staal and has a research interest in hematopoietic stem cells, their development into T cells and genetic defects that are associated with the loss of immune function.

The Staal lab is part of the Department of Immunohematology and Blood Transfusion (IHB), which is headed by Prof. Dr. W.E. Fibbe, MD, PhD. Our lab has shared work meetings with the lab of professor Fibbe and many collaborations within the IHB Department and other Departments in the LUMC (MCB, pediatrics, hematology, nephrology, embryology to name a few).

Gene Therapy of Immunodeficiencies

Current research is focused on the optimization and clinical translation of gene therapy of RAG1 deficiencies. This project brings together over 10 years of preclinical work and should result in application of lentiviral gene therapy of a codon optimized RAG1 in hematopoietic stem cells. In preclinical pipeline, we have also developed lentiviral gene therapy vectors for BTK deficiency and RAG2 deficiency.

Molecular signals in HSC

The hematopoietic stem cell (HSC) and its progeny is another topic of interest. Without understanding what makes a HSC grow, resulting in repopulation of the bone marrow and generation of the hematopoietic system, either during development or after a bone marrow transplantation, the development of new therapies would be difficult. The HSC research group has an interest in understanding which molecular signals make HSCs do what they do, which cells provide these signals and how they can be manipulated to improve transplantation outcomes. The lab currently focusses on Wnt and Notch signals and their effect on hematopoietic differentiation.

T cell development

The development of T cells is closely to HSC differentiation. Different from other hematopoietic lineages, T cells develop in the thymus after it has been seeded with T cell progenitors from the bone marrow. Our focus is on the understanding of the dynamics of hematopoietic clones that seed the thymus and subsequently differentiate into mature T cells. Our current work also involves the analysis of human immunodeficiencies in a xenotransplant model to determine at which point during T cell development different mutations cause arrests in development. In addition, we are determining how interference with Wnt signals causes T cell development arrests and lymphoid leukemia.

Lab members:

prof. dr. Frank Staal
dr. Karin Pike-Overzet
dr. Martijn Brugman
drs. Farbod Famili
drs. Jolanda de Roo
Edwin de Haas
Marja van Eggermond
Sandra Vloemans

The Staal lab in September 2011: left to right back to front Farbod Famili, Anna-Sophia Wiekmeijer, Tom van Leusden, Martijn Brugman, Paul Roozen, Alexander Troullioud Lucas, Sandra Bres-Vloemans, Edwin de Haas, Frank Staal, Gita Naber, Karin Pike-Overzet, Jolanda de Roo, Miranda Baert and Machteld Tiemessen.

Former Lab members:

drs. Anna-Sophia Wiekmeijer
dr. Machteld Tiemessen
dr. Sacha Brigitte Geutskens
drs. Paul Roozen
dr. Tiago Luis
dr. Floor Weerkamp
dr. Y.Y. Ng
Miranda Baert
Gita Naber
dr. Kim Brand
dr. Kim van der Weerd
Mark Rodijk
dr. Cindy J.M. Loomans
dr. Mascha van Noort

Visiting Scientists:

Lika Osugui (Universidade Federal de São Paulo, Brasil)
Paula Pontrelli (Italy)
Hélia Neves (Lisbon, Portugal)