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Page 1
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: negre o. 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.
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: negre o. Nature. 2010 Sep 16;467(7313):318-22. doi: 10.1038/nature09328. Nature. 2010. PMID: 20844535 Free PMC article.
Lentivirus vectors in β-thalassemia.
Payen E, Colomb C, Negre O, Beuzard Y, Hehir K, Leboulch P. Payen E, et al. Among authors: negre o. Methods Enzymol. 2012;507:109-24. doi: 10.1016/B978-0-12-386509-0.00006-5. Methods Enzymol. 2012. PMID: 22365771
Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient.
Tubsuwan A, Abed S, Deichmann A, Kardel MD, Bartholomä C, Cheung A, Negre O, Kadri Z, Fucharoen S, von Kalle C, Payen E, Chrétien S, Schmidt M, Eaves CJ, Leboulch P, Maouche-Chrétien L. Tubsuwan A, et al. Among authors: negre o. Stem Cells. 2013 Sep;31(9):1785-94. doi: 10.1002/stem.1436. Stem Cells. 2013. PMID: 23712774
An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia.
Dussiot M, Maciel TT, Fricot A, Chartier C, Negre O, Veiga J, Grapton D, Paubelle E, Payen E, Beuzard Y, Leboulch P, Ribeil JA, Arlet JB, Coté F, Courtois G, Ginzburg YZ, Daniel TO, Chopra R, Sung V, Hermine O, Moura IC. Dussiot M, et al. Among authors: negre o. Nat Med. 2014 Apr;20(4):398-407. doi: 10.1038/nm.3468. Epub 2014 Mar 23. Nat Med. 2014. PMID: 24658077 Free PMC article.
HSP70 sequestration by free α-globin promotes ineffective erythropoiesis in β-thalassaemia.
Arlet JB, Ribeil JA, Guillem F, Negre O, Hazoume A, Marcion G, Beuzard Y, Dussiot M, Moura IC, Demarest S, de Beauchêne IC, Belaid-Choucair Z, Sevin M, Maciel TT, Auclair C, Leboulch P, Chretien S, Tchertanov L, Baudin-Creuza V, Seigneuric R, Fontenay M, Garrido C, Hermine O, Courtois G. Arlet JB, et al. Among authors: negre o. Nature. 2014 Oct 9;514(7521):242-6. doi: 10.1038/nature13614. Epub 2014 Aug 24. Nature. 2014. PMID: 25156257
29 results