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

来源：

MOLECULAR THERAPY | 2023年 / 31卷 / 04期

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：

引用

页码：1 / 1

页数：1

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[31] Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy [J].

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