EFFICACY AND SAFETY OF A SINGLE DOSE OF EXAGAMGLOGENE AUTOTEMCEL FOR TRANSFUSION-DEPENDENT.-THALASSEMIA AND SEVERE SICKLE CELL DISEASE

被引:0
作者
Locatelli, Franco [1 ]
Frangoul, Haydar [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 C. [13 ]
Bobruff, Yael [14 ]
Simard, Chris [14 ]
Song, Yang [14 ]
Zhang, Lanju [14 ]
Sharma, Anjali [15 ]
Imren, Suzan [14 ]
Hobbs, Bill [14 ]
Grupp, Stephan [16 ]
机构
[1] Osped Pediat Bambino Gesu, Rome, Italy
[2] Childrens Hosp, Sarah Cannon Ctr Blood Canc, Tri Star Centennial, Nashville, TN USA
[3] Univ Regensburg, Regensburg, Germany
[4] Imperial Coll Healthcare NHS Trust, St Marys Hosp, London, England
[5] Univ Toronto, Hosp Sick Children, Toronto, ON, Canada
[6] Univ Milan, Milan, Italy
[7] Univ Paris, Necker Enfants Malades Hosp, Assistance Publ Hop Paris, AP HP, Paris, France
[8] Univ Athens, Athens, Greece
[9] Gemeinschaftsklin Mittelrhein, Koblenz, Germany
[10] Univ Illinois, Chicago, IL USA
[11] Cornell Univ, Joan & Sanford Weill Med Coll, New York, NY USA
[12] Boston Univ, Sch Med, Boston, MA USA
[13] UCSF Benioff Childrens Hosp, Oakland, CA USA
[14] Vertex Pharmaceut Inc, Boston, MA USA
[15] CRISPR Therapeut, Cambridge, England
[16] Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Philadelphia, PA USA
关键词
D O I
暂无
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
O005
引用
收藏
页码:16 / 17
页数:2
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