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Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo.
Schwarzwaelder K, Howe SJ, Schmidt M, Brugman MH, Deichmann A, Glimm H, Schmidt S, Prinz C, Wissler M, King DJ, Zhang F, Parsley KL, Gilmour KC, Sinclair J, Bayford J, Peraj R, Pike-Overzet K, Staal FJ, de Ridder D, Kinnon C, Abel U, Wagemaker G, Gaspar HB, Thrasher AJ, von Kalle C. Schwarzwaelder K, et al. Among authors: zhang f. J Clin Invest. 2007 Aug;117(8):2241-9. doi: 10.1172/JCI31661. J Clin Invest. 2007. PMID: 17671654 Free PMC article. Clinical Trial.
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: zhang f. 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.
Correction of SCID-X1 using an enhancerless Vav promoter.
Almarza E, Zhang F, Santilli G, Blundell MP, Howe SJ, Thornhill SI, Bueren JA, Thrasher AJ. Almarza E, et al. Among authors: zhang f. Hum Gene Ther. 2011 Mar;22(3):263-70. doi: 10.1089/hum.2010.119. Epub 2011 Feb 7. Hum Gene Ther. 2011. PMID: 20887212
Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning.
Gaspar HB, Bjorkegren E, Parsley K, Gilmour KC, King D, Sinclair J, Zhang F, Giannakopoulos A, Adams S, Fairbanks LD, Gaspar J, Henderson L, Xu-Bayford JH, Davies EG, Veys PA, Kinnon C, Thrasher AJ. Gaspar HB, et al. Among authors: zhang f. Mol Ther. 2006 Oct;14(4):505-13. doi: 10.1016/j.ymthe.2006.06.007. Epub 2006 Aug 14. Mol Ther. 2006. PMID: 16905365 Free article.
Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction.
Gaspar HB, Cooray S, Gilmour KC, Parsley KL, Zhang F, Adams S, Bjorkegren E, Bayford J, Brown L, Davies EG, Veys P, Fairbanks L, Bordon V, Petropoulou T, Kinnon C, Thrasher AJ. Gaspar HB, et al. Among authors: zhang f. Sci Transl Med. 2011 Aug 24;3(97):97ra80. doi: 10.1126/scitranslmed.3002716. Sci Transl Med. 2011. PMID: 21865538 Clinical Trial.
Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
Garcia-Perez L, van Eggermond M, van Roon L, Vloemans SA, Cordes M, Schambach A, Rothe M, Berghuis D, Lagresle-Peyrou C, Cavazzana M, Zhang F, Thrasher AJ, Salvatori D, Meij P, Villa A, Van Dongen JJM, Zwaginga JJ, van der Burg M, Gaspar HB, Lankester A, Staal FJT, Pike-Overzet K. Garcia-Perez L, et al. Among authors: zhang f. Mol Ther Methods Clin Dev. 2020 Mar 31;17:666-682. doi: 10.1016/j.omtm.2020.03.016. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32322605 Free PMC article.
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer.
Pike-Overzet K, Rodijk M, Ng YY, Baert MR, Lagresle-Peyrou C, Schambach A, Zhang F, Hoeben RC, Hacein-Bey-Abina S, Lankester AC, Bredius RG, Driessen GJ, Thrasher AJ, Baum C, Cavazzana-Calvo M, van Dongen JJ, Staal FJ. Pike-Overzet K, et al. Among authors: zhang f. Leukemia. 2011 Sep;25(9):1471-83. doi: 10.1038/leu.2011.106. Epub 2011 May 27. Leukemia. 2011. PMID: 21617701
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: zhang f. 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
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