OUTCOMES OF GENE THERAPY FOR BETA-THALASSEMIA MAJOR VIA TRANSPLANTATION OF AUTOLOGOUS HEMATOPOIETIC STEM CELLS TRANSDUCED EX VIVO WITH A LENTIVIRAL BETA GLOBIN VECTOR.

被引：0

作者：

Cavazzana, M. ^{[1
]}

Ribeil, J. A. ^{[1
]}

Payen, E. ^{[2
,3
]}

Suarez, F. ^{[4
]}

Negre, O. ^{[5
]}

Beuzard, Y. ^{[2
,3
]}

Touzot, F. ^{[1
]}

Cavallesco, R. ^{[6
,7
]}

Lefrere, F. ^{[1
]}

Chretien, S. ^{[2
,3
]}

Bourget, P. ^{[8
]}

Monpoux, F. ^{[9
]}

Pondarre, C. ^{[10
]}

Neven, B. ^{[11
]}

Bushman, F. D. ^{[12
]}

Schmidt, M. ^{[13
,14
]}

von Kalle, C. ^{[13
,14
]}

Sandler, L. ^{[15
]}

Soni, S. ^{[15
]}

Ryu, B. ^{[15
]}

Kutner, R. ^{[15
]}

Veres, G. ^{[15
]}

Finer, M. ^{[15
]}

Blanche, S. ^{[11
]}

Hermine, O. ^{[4
]}

Hacein-Bey-Abina, S. ^{[1
]}

Leboulch, P. ^{[2
,3
,6
,7
]}

机构：

[1] Hop Univ Necker Enfants Malad, Dept Biotherapie, Paris, France

[2] Univ Paris 11, INSERM, CEA, UMR 962, Orsay, France

[3] CEA, Inst Emerging Dis & Innovat Therapies iMETI, Fontenay Aux Roses, France

[4] Hop Univ Necker Enfants Malad, Serv Hematol Adulte, Paris, France

[5] CEA, IMETI, Bluebird Bio France, Fontenay Aux Roses, France

[6] Harvard Univ, Sch Med, Boston, MA USA

[7] Brigham & Womens Hosp, Boston, MA 02115 USA

[8] Hop Univ Necker Enfants Malad, Clin Pharm Dept, Paris, France

[9] Ctr Hosp Nice Sophia Antipolis, Unite Hematooncol Infantile, Nice, France

[10] CHIC, Ctr Reference Drepanocytose, Serv Pediat, Creteil, France

[11] Hop Univ Necker Enfants Malad, Unite Immunol Hematol Pediat, Paris, France

[12] Univ Penn, Dept Microbiol, Perelman Sch Med, Philadelphia, PA 19104 USA

[13] Natl Ctr Tumor Dis NCT, Heidelberg, Germany

[14] German Canc Res Ctr, Heidelberg, Germany

[15] Bluebird Bio, Cambridge, England

来源：

HAEMATOLOGICA | 2014年 / 99卷

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

S742

引用

页码：268 / 268

页数：1

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