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Gene therapy for primary immunodeficiencies.
Rivat C, Santilli G, Gaspar HB, Thrasher AJ. Rivat C, et al. Hum Gene Ther. 2012 Jul;23(7):668-75. doi: 10.1089/hum.2012.116. Hum Gene Ther. 2012. PMID: 22691036 Free PMC article. Review.
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S, Bleesing J, Blondeau J, de Boer H, Buckland KF, Caccavelli L, Cros G, De Oliveira S, Fernández KS, Guo D, Harris CE, Hopkins G, Lehmann LE, Lim A, London WB, van der Loo JC, Malani N, Male F, Malik P, Marinovic MA, McNicol AM, Moshous D, Neven B, Oleastro M, Picard C, Ritz J, Rivat C, Schambach A, Shaw KL, Sherman EA, Silberstein LE, Six E, Touzot F, Tsytsykova A, Xu-Bayford J, Baum C, Bushman FD, Fischer A, Kohn DB, Filipovich AH, Notarangelo LD, Cavazzana M, Williams DA, Thrasher AJ. Hacein-Bey-Abina S, et al. N Engl J Med. 2014 Oct 9;371(15):1407-17. doi: 10.1056/NEJMoa1404588. N Engl J Med. 2014. PMID: 25295500 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. 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.
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