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Page 1
Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.
Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K, Picard C, Six E, Himoudi N, Gilmour K, McNicol AM, Hara H, Xu-Bayford J, Rivat C, Touzot F, Mavilio F, Lim A, Treluyer JM, Héritier S, Lefrère F, Magalon J, Pengue-Koyi I, Honnet G, Blanche S, Sherman EA, Male F, Berry C, Malani N, Bushman FD, Fischer A, Thrasher AJ, Galy A, Cavazzana M. Hacein-Bey Abina S, et al. Among authors: malani n. JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253. JAMA. 2015. PMID: 25898053 Free PMC article. Clinical Trial.
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. Among authors: malani n. 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.
INSPIIRED: Quantification and Visualization Tools for Analyzing Integration Site Distributions.
Berry CC, Nobles C, Six E, Wu Y, Malani N, Sherman E, Dryga A, Everett JK, Male F, Bailey A, Bittinger K, Drake MJ, Caccavelli L, Bates P, Hacein-Bey-Abina S, Cavazzana M, Bushman FD. Berry CC, et al. Among authors: malani n. Mol Ther Methods Clin Dev. 2016 Dec 18;4:17-26. doi: 10.1016/j.omtm.2016.11.003. eCollection 2017 Mar 17. Mol Ther Methods Clin Dev. 2016. PMID: 28344988 Free PMC article.
INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes.
Sherman E, Nobles C, Berry CC, Six E, Wu Y, Dryga A, Malani N, Male F, Reddy S, Bailey A, Bittinger K, Everett JK, Caccavelli L, Drake MJ, Bates P, Hacein-Bey-Abina S, Cavazzana M, Bushman FD. Sherman E, et al. Among authors: malani n. Mol Ther Methods Clin Dev. 2016 Dec 18;4:39-49. doi: 10.1016/j.omtm.2016.11.002. eCollection 2017 Mar 17. Mol Ther Methods Clin Dev. 2016. PMID: 28344990 Free PMC article.
A method to sequence and quantify DNA integration for monitoring outcome in gene therapy.
Brady T, Roth SL, Malani N, Wang GP, Berry CC, Leboulch P, Hacein-Bey-Abina S, Cavazzana-Calvo M, Papapetrou EP, Sadelain M, Savilahti H, Bushman FD. Brady T, et al. Among authors: malani n. Nucleic Acids Res. 2011 Jun;39(11):e72. doi: 10.1093/nar/gkr140. Epub 2011 Mar 16. Nucleic Acids Res. 2011. PMID: 21415009 Free PMC article.
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