Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

被引：28

作者：

Kanter, Julie ^{[1
]}

Walters, Mark C. ^{[2
]}

Hsieh, Matthew M. ^{[3
]}

Krishnamurti, Lakshmanan ^{[4
,5
]}

Kwiatkowski, Janet ^{[6
]}

Kamble, Rammurti T. ^{[7
,8
]}

von Kalle, Christof ^{[9
]}

Kuypers, Frans A. ^{[10
]}

Cavazzana, Marina ^{[11
]}

Leboulch, Philippe ^{[12
,13
,14
,15
,16
]}

Joseney-Antoine, Marcelyne ^{[17
]}

Asmal, Mohammed ^{[17
]}

Thompson, Alexis A. ^{[18
]}

Tisdale, John F. ^{[3
]}

机构：

[1] Med Univ S Carolina, Div Pediat, Charleston, SC USA

[2] UCSF Benioff Childrens Hosp Oakland, Oakland, CA USA

[3] NHLBI, Mol & Clin Hematol Branch, NIDDK, NIH, Bethesda, MD USA

[4] Emory Univ, Aflac Canc & Blood Disorders Ctr, Atlanta, GA USA

[5] Childrens Healthcare Atlanta, Atlanta, GA USA

[6] Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA USA

[7] Baylor Coll Med, Ctr Cell & Gene Therapy, Houston, TX USA

[8] Houston Methodist Hosp, Houston, TX USA

[9] German Canc Res Ctr, Natl Ctr Tumor Dis NCT, Heidelberg, Germany

[10] Childrens Hosp, Oakland Res Inst, 747 52nd St, Oakland, CA 94609 USA

[11] Grp Hosp Univ Ouest, AP HP, Biotherapy Clin Invest Ctr, INSERM, Paris, France

[12] CEA, Fontenay Aux Roses, France

[13] Univ Paris Sud, Inst Emerging Dis & Innovat Therapies, Fontenay Aux Roses, France

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

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

[16] Brigham & Womens Hosp, Boston, MA USA

[17] Bluebird Bio, Cambridge, MA USA

[18] Ann Robert H Lurie Childrens Hosp Chicago, Div Hematol Oncol Stem Cell Transplant, Chicago, IL USA

来源：

关键词：

D O I：

10.1182/blood.V128.22.1176.1176

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

1176

引用

页数：5