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Gene therapy for inherited immunodeficiency.
Touzot F, Hacein-Bey-Abina S, Fischer A, Cavazzana M. Touzot F, et al. Expert Opin Biol Ther. 2014 Jun;14(6):789-98. doi: 10.1517/14712598.2014.895811. Epub 2014 Mar 8. Expert Opin Biol Ther. 2014. PMID: 24823313 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.
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. JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253. JAMA. 2015. PMID: 25898053 Free PMC article. Clinical Trial.
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.
Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. Clarke EL, et al. Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z. Genome Med. 2018. PMID: 30261899 Free PMC article.
Gene therapy for primary immunodeficiencies.
Fischer A, Hacein-Bey Abina S, Touzot F, Cavazzana M. Fischer A, et al. Clin Genet. 2015 Dec;88(6):507-15. doi: 10.1111/cge.12576. Epub 2015 Mar 23. Clin Genet. 2015. PMID: 25708106 Review.
Restoration of human B-cell differentiation into NOD-SCID mice engrafted with gene-corrected CD34+ cells isolated from Artemis or RAG1-deficient patients.
Lagresle-Peyrou C, Benjelloun F, Hue C, Andre-Schmutz I, Bonhomme D, Forveille M, Beldjord K, Hacein-Bey-Abina S, De Villartay JP, Charneau P, Durandy A, Fischer A, Cavazzana-Calvo M. Lagresle-Peyrou C, et al. Mol Ther. 2008 Feb;16(2):396-403. doi: 10.1038/sj.mt.6300353. Epub 2007 Nov 27. Mol Ther. 2008. PMID: 18223550 Free article.
111 results