Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

被引：0
|
作者：
Walters, Mark C. [1 ]
Rasko, John [2 ,3 ]
Hongeng, Suradej [4 ]
Kwiatkowski, Janet [5 ]
Schiller, Gary J. [6 ]
Kletzel, Morris [7 ]
Ho, P. Joy [8 ]
Vichinsky, Elliott [9 ]
von Kalle, Christof [10 ]
Cavazzana, Marina [11 ]
Leboulch, Philippe [4 ,12 ,13 ,14 ,15 ]
Petrusich, Alexandria [16 ]
Soni, Sandeep [16 ]
Thompson, Alexis A. [7 ]
机构：
[1] UCSF Benioff Childrens Hosp, Oakland, CA USA
[2] Royal Prince Alfred Hosp, Cell & Mol Therapies, Camperdown, NSW 2050, Australia
[3] Royal Prince Alfred Hosp, Inst Haematol, Camperdown, NSW 2050, Australia
[4] Mahidol Univ, Ramathibodi Hosp, Bangkok 10700, Thailand
[5] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[6] Univ Calif Los Angeles, Los Angeles, CA USA
[7] Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA
[8] Royal Prince Alfred Hosp, Inst Haematol, Sydney, NSW, Australia
[9] UCSF Benioff Childrens Hosp Oakland, Hematol Oncol, Oakland, CA USA
[10] German Canc Res Ctr, Natl Ctr Tumor Dis NCT, Heidelberg, Germany
[11] Hop Univ Necker Enfants Malad, Biotherapy Dept, Paris, France
[12] Brigham & Womens Hosp, Boston, MA 02115 USA
[13] Harvard Univ, Sch Med, Boston, MA USA
[14] CEA IMETI, Fontenay Aux Roses, France
[15] Univ Paris 11, Fontenay Aux Roses, France
[16] Bluebird Bio Inc, Cambridge, MA USA
来源：
BLOOD | 2015年 / 126卷 / 23期
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中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
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页数：3
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