Efficacy and safety of LentiGlobin gene therapy in patients with transfusion-dependent β-thalassaemia and non-β0/β0 genotypes: Updated results from the completed phase 1/2 Northstar and ongoing phase 3 Northstar-2 studies

被引：0

作者：

Porter, J. B. ^{[1
]}

Walters, M. C. ^{[2
]}

Kwiatkowski, J. L. ^{[3
]}

Hongeng, S. ^{[4
]}

Rasko, J. E. J. ^{[5
]}

Sauer, M. G. ^{[6
]}

Thrasher, A. J. ^{[7
]}

Thuret, I. ^{[8
]}

Schiller, G. J. ^{[9
]}

Elliot, H. ^{[10
]}

Deary, B. ^{[10
]}

Chen, Y. ^{[10
]}

Tao, G. ^{[10
]}

Asmal, M. ^{[10
]}

Locatelli, F. ^{[11
]}

Thompson, A. A. ^{[12
]}

机构：

[1] Univ Coll London Hosp, Haematol Dept, London, England

[2] UCSF Benioff Childrens Hosp Oakland, Hematol Oncol BMT, Oakland, CA USA

[3] Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA

[4] Mahidol Univ, Ramathibodi Hosp, Bangkok, Thailand

[5] Royal Prince Alfred Hosp, Dept Cell & Mol Therapies, Camperdown, NSW, Australia

[6] Hannover Med Sch, Pediat Hematol Oncol Blood Cell Transplantat, Hannover, Germany

[7] UCL Great Ormond St Inst Child Hlth, London, England

[8] Hop La Timone, Pediat Hematol, Marseille, France

[9] UCLA Med, David Geffen Sch Med, Hematol Oncol, Los Angeles, CA USA

[10] Bluebird Bio Inc, Cambridge, MA USA

[11] IRCCS Bambino Gesu Childrens Hosp, Dept Pediat Hematol Oncol & Cell & Gene Therapy, Rome, Italy

[12] Ann & Robert H Lurie Childrens Hosp, Pediat Hematol, Chicago, IL USA

来源：

关键词：

autologous stem cell transplant; beta-thalassemia; gene therapy;

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

BSH19-OR-0

引用

页码：23 / 23

页数：1