Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

被引:0
作者
Frangoul, Haydar [1 ]
Locatelli, Franco [2 ]
Corbacioglu, Selim [3 ]
De La Fuente, Josu [4 ]
Wall, Donna [5 ]
Cappellini, Maria Domenica [6 ]
de Montalembert, Mariane [7 ]
Kattamis, Antonis [8 ]
Lobitz, Stephan [9 ]
Rondelli, Damiano [10 ]
Sheth, Sujit [11 ]
Steinberg, Martin [12 ]
Walters, Mark [13 ]
Bobruff, Yael [14 ]
Simard, Chris [14 ]
Song, Yang [14 ]
Zhang, Lanju [14 ]
Sharma, Anjali [15 ]
Imren, Suzan [14 ]
Hobbs, Bill [14 ]
Grupp, Stephan A. [16 ]
机构
[1] Childrens Hosp TriStar Centennial, Nashville, TN USA
[2] Uni Rome, Rome, Italy
[3] Uni Regensburg, Regensburg, Germany
[4] Imperial Coll London, St Albans, England
[5] Hosp Sick Children, Toronto, ON, Canada
[6] Uni Milan, Milan, Italy
[7] Uni Paris, Paris, France
[8] Uni Athens, Athens, Greece
[9] Gemeinschaftsklinikum Mittelrhein, Koblenz, Germany
[10] Uni Ill Chicago, Chicago, IL USA
[11] Weill Cornell Med, New York, NY USA
[12] BU Sch Med, Boston, MA USA
[13] UCSF Benioff Childrens Hosp, Oakland, CA USA
[14] Vertex Pharmaceut, Boston, MA USA
[15] CRISPR Therapeut, Cambridge, MA USA
[16] Uni Penn, Perelman Sch Med, Philadelphia, PA USA
关键词
D O I
暂无
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
1
引用
收藏
页码:1 / 1
页数:1
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