![]() Hendriks RW, Bredius RG, Pike-Overzet K, Staal FJ.Įxpert Opin Ther Targets. PMID: 22430378 Biology and novel treatment options for XLA, the most common monogenetic immunodeficiency in man. PMID: 24940562 Induced pluripotent stem cells and severe combined immunodeficiency: merely disease modeling or potentially a novel cure? Wiekmeijer AS, Pike-Overzet K, Brugman MH, Salvatori DC, Egeler RM, Bredius RG, Fibbe WE, Staal FJ.īiores Open Access. PMID: 25117803 Sustained Engraftment of Cryopreserved Human Bone Marrow CD34(+) Cells in Young Adult NSG Mice. Pike-Overzet K, Baum C, Bredius RG, Cavazzana M, Driessen GJ, Fibbe WE, Gaspar HB, Hoeben RC, Lagresle-Peyrou C, Lankester A, Meij P, Schambach A, Thrasher A, Van Dongen JJ, Zwaginga JJ, Staal FJ. PMID: 26441229 Successful RAG1-SCID gene therapy depends on the level of RAG1 expression. Relevant publications: Identification of checkpoints in human T-cell development using severe combined immunodeficiency stem cells.Īnna-Sophia Wiekmeijer, Karin Pike-Overzet, Martijn H Brugman, Ingrid LM Wolvers-Tettero, Arjan C Lankester, Robbert GM Bredius, Jacques JM van Dongen, Willem E Fibbe, Anton W Langerak, Mirjam van der Burg, Frank JT Staal mRNA expression measurements (qPCR, microarray, RNASeq).Retro- or lentiviral expression of transgenes.Models for transplantation of human stem cells (NSG).Genetic or conditional knockout mouse models (Rag1, Rag2, Btk). ![]() ![]() We closely collaborate with the Willem Alexander Children Clinic at LUMC and the patient advocacy group Stichting afweer stoornissen. Soon you can find updates here with information for patients and their parents. Cossu, Italian Journal of Pediatrics 2010. Currently we are close to clinical implementation of gene therapy for RAG1-deficient SCID. Our main emphasis is on treatment of severe combined immune deficiency (SCID), sometimes called bubble boy disease, and other primary immune deficiencies such as XLA (BTK-disease). In our lab, we focus on gene therapy for a variety of immune disorders. Gene therapy refers to the transfer of genetic information with the intend to cure a diases.
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