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：

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：

引用

页数：3