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Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.
Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G. Huston MW, et al. Among authors: visser tp. Mol Ther. 2011 Oct;19(10):1867-77. doi: 10.1038/mt.2011.127. Epub 2011 Jul 12. Mol Ther. 2011. PMID: 21750532 Free PMC article.
Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype.
van Til NP, Stok M, Aerts Kaya FS, de Waard MC, Farahbakhshian E, Visser TP, Kroos MA, Jacobs EH, Willart MA, van der Wegen P, Scholte BJ, Lambrecht BN, Duncker DJ, van der Ploeg AT, Reuser AJ, Verstegen MM, Wagemaker G. van Til NP, et al. Among authors: visser tp. Blood. 2010 Jul 1;115(26):5329-37. doi: 10.1182/blood-2009-11-252874. Epub 2010 Apr 12. Blood. 2010. PMID: 20385789 Free article.
Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome.
van Til NP, Sarwari R, Visser TP, Hauer J, Lagresle-Peyrou C, van der Velden G, Malshetty V, Cortes P, Jollet A, Danos O, Cassani B, Zhang F, Thrasher AJ, Fontana E, Poliani PL, Cavazzana M, Verstegen MM, Villa A, Wagemaker G. van Til NP, et al. Among authors: visser tp. J Allergy Clin Immunol. 2014 Apr;133(4):1116-23. doi: 10.1016/j.jaci.2013.10.009. Epub 2013 Dec 9. J Allergy Clin Immunol. 2014. PMID: 24332219
Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease.
Stok M, de Boer H, Huston MW, Jacobs EH, Roovers O, Visser TP, Jahr H, Duncker DJ, van Deel ED, Reuser AJJ, van Til NP, Wagemaker G. Stok M, et al. Among authors: visser tp. Mol Ther Methods Clin Dev. 2020 May 4;17:1014-1025. doi: 10.1016/j.omtm.2020.04.023. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32462050 Free PMC article.
Stimulation of mouse bone marrow cells with kit ligand, FLT3 ligand, and thrombopoietin leads to efficient retrovirus-mediated gene transfer to stem cells, whereas interleukin 3 and interleukin 11 reduce transduction of short- and long-term repopulating cells.
Wognum AW, Visser TP, Peters K, Bierhuizen MF, Wagemaker G. Wognum AW, et al. Among authors: visser tp. Hum Gene Ther. 2000 Oct 10;11(15):2129-41. doi: 10.1089/104303400750001435. Hum Gene Ther. 2000. PMID: 11044914
Keratinocyte growth factor and stem cell factor to improve thymopoiesis after autologous CD34+ cell transplantation in rhesus macaques.
Wils EJ, Aerts-Kaya FS, Rombouts EJ, van Mourik I, Rijken-Schelen A, Visser TP, Braakman E, Wagemaker G, Cornelissen JJ. Wils EJ, et al. Among authors: visser tp. Biol Blood Marrow Transplant. 2012 Jan;18(1):55-65. doi: 10.1016/j.bbmt.2011.09.010. Epub 2011 Oct 1. Biol Blood Marrow Transplant. 2012. PMID: 21963880 Free article.
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