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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: blanche s. 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.
Impact of HLA matching on outcome of hematopoietic stem cell transplantation in children with inherited diseases: a single-center comparative analysis of genoidentical, haploidentical or unrelated donors.
Caillat-Zucman S, Le Deist F, Haddad E, Gannagé M, Dal Cortivo L, Jabado N, Hacein-Bey-Abina S, Blanche S, Casanova JL, Fischer A, Cavazzana-Calvo M. Caillat-Zucman S, et al. Among authors: blanche s. Bone Marrow Transplant. 2004 Jun;33(11):1089-95. doi: 10.1038/sj.bmt.1704510. Bone Marrow Transplant. 2004. PMID: 15077132
Failure of SCID-X1 gene therapy in older patients.
Thrasher AJ, Hacein-Bey-Abina S, Gaspar HB, Blanche S, Davies EG, Parsley K, Gilmour K, King D, Howe S, Sinclair J, Hue C, Carlier F, von Kalle C, de Saint Basile G, le Deist F, Fischer A, Cavazzana-Calvo M. Thrasher AJ, et al. Among authors: blanche s. Blood. 2005 Jun 1;105(11):4255-7. doi: 10.1182/blood-2004-12-4837. Epub 2005 Feb 1. Blood. 2005. PMID: 15687233 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: blanche s. J Clin Invest. 2008 Sep;118(9):3132-42. doi: 10.1172/JCI35700. J Clin Invest. 2008. PMID: 18688285 Free PMC article. Clinical Trial.
Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency.
Neven B, Leroy S, Decaluwe H, Le Deist F, Picard C, Moshous D, Mahlaoui N, Debré M, Casanova JL, Dal Cortivo L, Madec Y, Hacein-Bey-Abina S, de Saint Basile G, de Villartay JP, Blanche S, Cavazzana-Calvo M, Fischer A. Neven B, et al. Among authors: blanche s. Blood. 2009 Apr 23;113(17):4114-24. doi: 10.1182/blood-2008-09-177923. Epub 2009 Jan 23. Blood. 2009. PMID: 19168787 Free article.
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: blanche s. Science. 2009 Nov 6;326(5954):818-23. doi: 10.1126/science.1171242. Science. 2009. PMID: 19892975
557 results