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Page 1
Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M. Ribeil JA, et al. Among authors: caccavelli l. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677. N Engl J Med. 2017. PMID: 28249145 Free article.
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, Clappier E, Caccavelli L, Delabesse E, Beldjord K, Asnafi V, MacIntyre E, Dal Cortivo L, Radford I, Brousse N, Sigaux F, Moshous D, Hauer J, Borkhardt A, Belohradsky BH, Wintergerst U, Velez MC, Leiva L, Sorensen R, Wulffraat N, Blanche S, Bushman FD, Fischer A, Cavazzana-Calvo M. Hacein-Bey-Abina S, et al. Among authors: caccavelli l. J Clin Invest. 2008 Sep;118(9):3132-42. doi: 10.1172/JCI35700. J Clin Invest. 2008. PMID: 18688285 Free PMC article. Clinical Trial.
Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, Mahlaoui N, Kiermer V, Mittelstaedt D, Bellesme C, Lahlou N, Lefrère F, Blanche S, Audit M, Payen E, Leboulch P, l'Homme B, Bougnères P, Von Kalle C, Fischer A, Cavazzana-Calvo M, Aubourg P. Cartier N, et al. Among authors: caccavelli l, l homme b. Science. 2009 Nov 6;326(5954):818-23. doi: 10.1126/science.1171242. Science. 2009. PMID: 19892975
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.
Cavazzana-Calvo M, Payen E, Negre O, Wang G, Hehir K, Fusil F, Down J, Denaro M, Brady T, Westerman K, Cavallesco R, Gillet-Legrand B, Caccavelli L, Sgarra R, Maouche-Chrétien L, Bernaudin F, Girot R, Dorazio R, Mulder GJ, Polack A, Bank A, Soulier J, Larghero J, Kabbara N, Dalle B, Gourmel B, Socie G, Chrétien S, Cartier N, Aubourg P, Fischer A, Cornetta K, Galacteros F, Beuzard Y, Gluckman E, Bushman F, Hacein-Bey-Abina S, Leboulch P. Cavazzana-Calvo M, et al. Among authors: caccavelli l. Nature. 2010 Sep 16;467(7313):318-22. doi: 10.1038/nature09328. Nature. 2010. PMID: 20844535 Free PMC article.
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: caccavelli l. 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.
Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1.
Touzot F, Moshous D, Creidy R, Neven B, Frange P, Cros G, Caccavelli L, Blondeau J, Magnani A, Luby JM, Ternaux B, Picard C, Blanche S, Fischer A, Hacein-Bey-Abina S, Cavazzana M. Touzot F, et al. Among authors: caccavelli l. Blood. 2015 Jun 4;125(23):3563-9. doi: 10.1182/blood-2014-12-616003. Epub 2015 Apr 13. Blood. 2015. PMID: 25869287 Free 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. Among authors: caccavelli l. JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253. JAMA. 2015. PMID: 25898053 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: caccavelli l. 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: caccavelli l. 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.
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Thompson AA, et al. Among authors: caccavelli l. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342. N Engl J Med. 2018. PMID: 29669226 Free article. Clinical Trial.
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